Revumenib

– U.S. launch expected in early February – – Niktimvo is the first and only approved treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis – – Pivotal data from the AGAVE-201 trial supporting FDA approval show treatment with Niktimvo resulted in durable responses across all organs studied and patient subgroups – WILMINGTON, Del. and WALTHAM, Mass., Jan. 15, 2025 /PRNewswire/ -- Incyte (Nasdaq:INCY) and Syndax Pharmaceuticals (Nasdaq:SNDX) today announced that the U.S. Food and Drug Administration (FDA) has approved Niktimvo™ (axatilimab-csfr) in 9 mg and 22 mg vial sizes. The Companies expect product to be available for order in the U.S. in early February. Niktimvo is approved for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Niktimvo is the first and only FDA-approved prescription treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis. "We are thrilled to build on our strong commitment to the GVHD community with the U.S. launch of Niktimvo, a first-in-class therapeutic agent that has demonstrated remarkable responses in patients with chronic GVHD whose response was suboptimal after at least two prior lines of systemic therapy," said Hervé Hoppenot, Chief Executive Officer, Incyte. "Our deep understanding of chronic GVHD and our connections in the clinical community will support a successful launch, in partnership with Syndax, of this important medicine for patients." Niktimvo was approved by the FDA on August 14, 2024. The approval was based on positive data from the global AGAVE-201 trial, which were published in the New England Journal of Medicine in September 2024.1 The trial met the primary endpoint across all cohorts receiving Niktimvo with 75% of patients who received 0.3 mg/kg every two weeks achieving a response at six months of treatment (N=79). "As the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD, Niktimvo represents a major breakthrough for patient care," said Michael Metzger, Chief Executive Officer, Syndax. "Together with Incyte, we look forward to executing a robust commercial launch and advancing the treatment paradigm for patients with chronic GVHD who have progressed after at least two lines of systemic therapy." Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in > 2 patients included infection (pathogen unspecified) (14%), viral infection (14%), and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain and pyrexia. The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia and dyspnea. The approved dose of Niktimvo for adults and pediatric patients weighing at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks. Niktimvo will be available for healthcare providers to order through a network of specialty distributors in both 9 mg vial and 22 mg vial sizes to facilitate patient dosing. Incyte and Syndax are committed to supporting patients and removing barriers to ensure access to Niktimvo. Eligible patients in the U.S. who are prescribed Niktimvo have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a comprehensive program offering personalized patient support, including financial assistance for eligible patients and ongoing education and additional resources. More information about IncyteCARES is available by visiting or calling 1-855-452-5234. On August 30, 2024, axatilimab-csfr (Niktimvo) was added to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a category 2A recommendation for the treatment of chronic GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.2 Treatments are classified as category 2A when there is uniform NCCN consensus that the intervention is appropriate, based on lower-level evidence. The updated NCCN guidelines are available at . In the U.S., Incyte and Syndax are co-commercializing Niktimvo. Incyte has exclusive commercialization rights for Niktimvo outside of the U.S. About Chronic Graft-Versus-Host Disease (GVHD) Chronic GVHD is a serious condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the transplant recipient's organs. Chronic GVHD is a leading cause of significant morbidity and mortality after an allogeneic stem cell transplant and is estimated to develop in approximately 42% of transplant recipients, affecting approximately 17,000 patients in the U.S.3 Of those patients who develop chronic GVHD, nearly 50% require at least three lines of treatment, emphasizing the need for additional effective treatment options.4 About AGAVE-201 The global AGAVE-201 dose-ranging trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD (GVHD) whose disease had progressed after two or more prior therapies. Patients were randomized to one of three treatment groups that investigated a distinct dose of axatilimab administered at 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks or 3.0 mg/kg every four weeks. The trial's primary endpoint was the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints included duration of response, percent reduction in daily steroid dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale. For more information about AGAVE-201, visit . About Niktimvo™ ( axatilimab-csfr) Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications. Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256). Niktimvo is a trademark of Incyte. All other trademarks are the property of their respective owners. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Infusion-Related Reactions Niktimvo™ (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received Niktimvo in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%. Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction. Embryo-Fetal Toxicity Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose. ADVERSE REACTIONS Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in >2 patients included infection (pathogen unspecified) (14%), viral infection (14%) and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia. The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea. Clinically relevant adverse reactions in <10% of patients who received Niktimvo included: Eye disorders: periorbital edema Skin and subcutaneous skin disorders: pruritus Vascular disorders: hypertension Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb. USE IN SPECIFIC POPULATIONS Lactation Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo. Females and Males of Reproductive Potential Pregnancy Testing Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo. Contraception Females Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo. DOSAGE AND ADMINISTRATION Dosage Modifications for Adverse Reactions Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations. Please see the full Prescribing Information for Niktimvo. About Incyte A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia. For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube. About Syndax Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit or follow the Company on X (formerly Twitter) and LinkedIn. Incyte Forward-Looking Statements Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding whether Niktimvo might provide a successful treatment option for patients with chronic GVHD: expectations regarding the launch of Niktimvo; and the potential for axatilimab to treat additional conditions, contain predictions, estimates and other forward-looking statements. These forward-looking statements are based on Incyte's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the U.S. FDA and other regulatory authorities outside of the U.S.; the efficacy or safety of Incyte and its partners' products; the acceptance of Incyte and its partners' products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in Incyte's reports filed with the Securities and Exchange Commission, including its annual report on form 10-Kand its report on form 10-Q for the quarter ended September 30, 2024. Incyte disclaims any intent or obligation to update these forward-looking statements. Syndax Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend," "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of our product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Niktimvo's commercial availability; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. ­­­­­­1 Wolff D, et al. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease. N Engl J Med 2024;391:1002-14. DOI: 10.1056/NEJMoa2401537. 2 NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Hematopoietic Cell Transplantation (HCT). Version 2.2024 – August 30, 2024. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 3 Data on file. 4 Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A U.S. Claims Analysis. 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2024’s novel drug approvals offered a wide array of new cancer drugs, rare disease treatments, a potential Alzheimer’s blockbuster, and about 10 fewer approvals than 2023. While 2022 and 2023 saw major approvals of new cell and gene therapies, 2024 did not stick as cleanly to a theme — instead featuring a grab-bag of long-awaited approvals. That included Eli Lilly’s amyloid-targeted Alzheimer’s drug, the first two treatments for the rare Niemann-Pick disease, and the first-ever mesenchymal stromal cell therapy. Several approvals in 2024 also deployed new ways to treat familiar diseases. For instance, Pfizer’s new hemophilia A or B drug Hympavzi is the first to target a particular protein in the blood-clotting process. Bristol Myers Squibb’s Cobenfy, approved for schizophrenia, is the first antipsychotic that targets cholinergic receptors and not dopamine receptors. What to watch for in 2025: more cancer drug approvals and major label expansions. 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As with the many checkpoint drugs already on the market, BeiGene hopes esophageal cancer will be the first of many indications Tevimbra will eventually be approved to treat, both on its own and in combination with other medicines. The drug was first approved in China in 2019. — Amber Tong Active ingredient: atidarsagene autotemcel Indication: Children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy Snapshot : With a $4.25 million price tag, Lenmeldy is on the list of the world’s most expensive medicines. The gene therapy is for an ultra-rare and fatal disease called early-onset metachromatic leukodystrophy, which affects fewer than 40 children a year in the US. Lenmeldy was already approved under a different brand name in the EU and UK and was shown in clinical trials to keep the majority of children who received it alive and with normal cognitive development. 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It was approved for children 6 months of age and up with a form of slow-growing brain cancer called low-grade glioma. “We now have shown that you can develop drugs intentionally for children at the speed of biotech,” Day One’s then-R&D chief Sam Blackman told Endpoints around Ojemda’s approval. — Lei Lei Wu Active ingredient: pivmecillinam Indication: Uncomplicated urinary tract infections caused by susceptible isolates of Escherichia coli , Proteus mirabilis and Staphylococcus saprophyticus , in female patients 18 years and older Snapshot : In April, Utility Therapeutics’ Pivya became the first new treatment for uncomplicated urinary tract infections in more than two decades. The oral antibiotic induced a composite response in 62% of patients versus just 10% for placebo in one of Utility’s three registrational trials. The UK-based drugmaker bought the US rights to pivmecillinam — the active ingredient in Pivya — from Leo Pharma as part of a 2018 deal with undisclosed financial terms. — Ayisha Sharma Active ingredient: fidanacogene elaparvovec-dzkt Indication: Moderate-to-severe hemophilia B in adults Snapshot : Pfizer became the second company to nab FDA approval for a one-time gene therapy to treat hemophilia B. And just like the US market’s first entrant, CSL, Pfizer priced the treatment at an eye-popping $3.5 million. Pfizer’s Beqvez was found to eliminate bleeding in 60% of patients in an open-label Phase 3 study, compared to 29% of patients receiving prophylaxis with a factor IX inhibitor. While it won approval for Beqvez, Pfizer has taken a step back from the gene therapy field, selling early assets in 2023 and dropping its Duchenne gene therapy after a failed trial. — Lei Lei Wu Active ingredient: mavorixafor Indication: WHIM syndrome in patients 12 and older Snapshot : Xolremdi was approved for an ultra-rare immune disorder, WHIM, named for its symptoms: warts, hypogammaglobulinemia, infections, and myelokathexis. X4 Pharmaceuticals’ drug is a CXCR4 inhibitor that is meant to address the underlying cause of the disease and improve patients’ ability to fight infections. X4 is also exploring Xolremdi’s use in treating chronic neutropenia. — Lei Lei Wu Active ingredient: tarlatamab-dlle Indication: Extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy. 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That includes 10 strains in common with Pfizer’s rival 20-valent shot Prevnar 20, plus 11 unique strains. — Nicole DeFeudis Active ingredient: sofpironium Indication: Primary axillary hyperhidrosis in adult and pediatric patients 9 years of age and older Snapshot : Botanix Pharmaceuticals unlocked a potential market of 10 million people in the US with the June approval of sofpironium for primary axillary hyperhidrosis, or excessive underarm sweat. Sold as a gel with the brand name Sofdra, Botanix launched an early patient experience program in the third quarter, followed by a broader launch in the fourth quarter, which is when it also expected its first revenues from the product. — Ayisha Sharma Active ingredient: crovalimab-akkz Indication: Adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria and body weight of at least 40 kg Snapshot : The FDA approval for Genentech’s C5 inhibitor crovalimab for people with paroxysmal nocturnal hemoglobinuria in June came with a significant caveat — the drug can only be given under a REMS pathway due to the serious risk of meningococcal infections. Two months after its US approval, crovalimab, sold as PiaSky, secured an EU green light for the rare disease. When left untreated, PNH causes intravascular hemolysis, thromboembolic events and cytopenia. — Ayisha Sharma Active ingredient: ensifentrine Indication: Maintenance treatment of chronic obstructive pulmonary disease in adult patients Snapshot : Verona Pharma launched its COPD drug Ohtuvayre in 2024, taking a step into a titan-filled arena. Known generically as ensifentrine, Ohtuvayre is a maintenance treatment for COPD, taken via nebulizer twice a day, that can be added to standard COPD therapies. Verona aims to carve out a niche for Ohtuvayre as an add-on treatment rather than competing directly with major pharma companies in COPD like GSK and AstraZeneca. — Lei Lei Wu Active ingredient: donanemab Indication: Alzheimer’s disease Snapshot : Eli Lilly has invested more than $8 billion over more than three decades in its search for an Alzheimer’s drug. It finally found success in July when the FDA approved Kisunla . Kisunla is the second amyloid-targeting antibody to get full FDA approval after Eisai and Biogen won approval for Leqembi last year. Experts once worried about Alzheimer’s drugs bankrupting Medicare, but sales of the drugs are off to a slower-than-expected start , partly due to the risk of potentially fatal brain bleeding and swelling — which occurred in about 1 in 300 people in Lilly’s study. Lilly hopes the drug will be safer and more effective when taken earlier, before symptoms of dementia begin. A large study testing that hypothesis is underway and is expected to wrap up in 2027. — Ryan Cross Active ingredient: deuruxolitinib Indication: Adults with severe alopecia areata Snapshot : Sun Pharma’s $576 million acquisition of Concert Pharmaceuticals in 2023 paid off with the approval of the JAK inhibitor Leqselvi in severe alopecia areata. Sun’s drug could compete against some big players, including Eli Lilly’s JAK inhibitor Olumiant and Pfizer’s Litfulo. However, the drug’s launch was delayed after Incyte, which makes a similar drug, secured an injunction against its sale in November. Sun said it planned to appeal. — Katherine Lewin Active ingredient: benzgalantamine Indication: Mild to moderate dementia of the Alzheimer’s type in adults Snapshot : Zunveyl is a new twist on an old pill that’s commonly used to help reduce some symptoms of Alzheimer’s disease, but potentially with fewer gastrointestinal side effects. The compound is a prodrug of galantamine, an acetylcholinesterase inhibitor approved in 2001 that blocks breakdown of a key neurochemical that brain cells use to communicate with each other. Alpha Cognition, a tiny Canadian company that won approval for Zunveyl in July based on bioavailability studies, said it will begin selling Zunveyl in early 2025. — Ryan Cross Active ingredient: afamitresgene autoleucel Indication: Unresectable or metastatic synovial sarcoma in adults have received prior chemotherapy, are HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive, and whose tumor expresses the MAGE-A4 antigen as determined by FDA authorized companion diagnostic devices. Snapshot : Tecelra became the first cell therapy based on T cell receptors and the second cell therapy for a solid tumor cancer to win approval in the US in 2024. The therapy, known as afami-cel, is meant for a subsection of synovial sarcoma patients whose cancer has spread throughout their body or can’t be removed via surgery, and who have already received chemotherapy. Patients need to be tested for expression of a protein called MAGE-A4, which is found in a range of tumors, as well as an immune marker called HLA-A*02. — Lei Lei Wu Active ingredient: vorasidenib Indication: For adult and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection. Snapshot : Brain cancer is getting its targeted therapies, too. Servier’s Voranigo was approved for people with slow-growing gliomas that bear an IDH mutation. Patients are eligible for the drug after surgery for their brain tumor. Over 80% of grade 2 gliomas bear an IDH mutation, and the FDA said this was the first approved systemic drug for this subset of patients. In a clinical trial versus placebo, the treatment staved off cancer progression for a median 16.6 months longer, which physicians called a “striking” result. — Lei Lei Wu Active ingredient: denileukin diftitox-cxdl Indication: Adult patients with relapsed or refractory stage I-III cutaneous T cell lymphoma after at least one prior systemic therapy Snapshot : Following a delay from a CRL over manufacturing issues in 2023, Lymphir gained FDA approval in August. In order to support the launch of the treatment for a rare cancer, the company’s cancer subsidiary, Citius Oncology, combined with TenX Keane Acquisition. — Katherine Lewin Active ingredient: palopegteriparatide Indication: Hypoparathyroidism in adults Snapshot : Ascendis rebounded from an earlier FDA rejection and subsequent delay of its new application to snag FDA approval of hypoparathyroidism drug Yorvipath. It was already approved in Austria and Germany, generating €1.5 million ($1.6 million) in the first quarter. Yorvipath’s US entry replaces a void left by Takeda’s Natpara, some two years after it was pulled off the market. Ascendis said at the time of approval that it was completing manufacturing of the product and expecting the first batch of supply to be available in the first quarter of 2025. — Max Bayer Active ingredient: nemolizumab Indication: Prurigo nodularis in adults Snapshot : In August, Galderma secured first-in-class status with the US approval of its IL-31 antibody nemolizumab for prurigo nodularis. The green light is the first step in unlocking Nemluvio’s blockbuster potential, much of which lies in eczema, according to Galderma. The Swiss drugmaker earned a label expansion for moderate-to-severe atopic dermatitis in December, with Jefferies analysts projecting $2.1 billion in global peak sales for the drug. — Ayisha Sharma Active ingredient: seladelpar Indication: Primary biliary cholangitis in combination with ursodeoxycholic acid, in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA Snapshot : After acquiring seladelpar in the $4.3 billion purchase of CymaBay Therapeutics, Gilead snagged FDA accelerated approval to treat PBC, boosting its liver portfolio. The drug joins three others on the market, though its ability to treat itch symptoms gives Gilead hope that it can edge out competitors. That potential benefit has also contributed to Livdelzi’s price, which is slightly higher than the other two approved therapies. — Max Bayer Active ingredient: axatilimab-csfr Indication: Chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg Snapshot : Thanks to the help of Syndax Pharmaceuticals, Incyte was able to get another cGVHD treatment approval in August. It already markets Jakafi. The newly approved CSF-1 receptor-blocking antibody will also be studied alongside Jakafi and in combination with steroids. Incyte has said it plans to target the approximately 6,500 people who have failed two prior drugs. It also hopes to get the medicine greenlit for idiopathic pulmonary fibrosis. — Kyle LaHucik Active ingredient: lazertinib Indication: Locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test Snapshot : Lazcluze was approved in August in combination with Johnson & Johnson’s Rybrevant for certain non-small cell lung cancer patients with two common EGFR mutations. The approval puts the combination therapy in direct competition with AstraZeneca and its blockbuster Tagrisso, which has been approved in the same lung cancer population since 2018. Tagrisso also recently won a label expansion as part of a chemotherapy combination regimen in that indication. — Nicole DeFeudis Active ingredient: lebrikizumab Indication: Moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable, in adult and pediatric patients 12 years and older who weigh at least 40 kg Snapshot : Following an FDA rejection over manufacturing issues in 2023 (and no concerns with safety or labeling), Lilly scored approval for Ebglyss in September for moderate-to-severe eczema. Between two studies, 38% of patients on Lilly’s drug achieved clear or almost-clear skin at 16 weeks. — Katherine Lewin Active ingredient: arimoclomol Indication: Neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 and older, in combination with miglustat Snapshot : Zevra Therapeutics won approval for Miplyffa, known generically as arimoclomol, for people with the rare genetic disorder Niemann-Pick disease type C who are aged 2 years and older. Miplyffa’s approval came after the FDA previously rejected the drug in 2021, but Zevra returned to ask for approval with a reanalysis of clinical trial data that showed improvements in swallowing, which can be affected by the disease. FDA hosted an advisory committee ahead of its decision that voted in favor of the drug for the disease, a lysosomal storage disorder in which the body can’t properly recycle certain fatty substances. — Lei Lei Wu Active ingredient: levacetylleucine Indication: Neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing ≥15 kg Snapshot : In September, IntraBio’s levacetylleucine became the second drug to clinch FDA approval for Niemann-Pick disease type C in the span of just one week. Branded Aqneursa, the oral drug can be taken up to three times daily with weight-dependent dosing. — Ayisha Sharma Active ingredient: xanomeline and trospium chloride Indication: Schizophrenia in adults Snapshot : Cobenfy’s approval gives Bristol Myers a significant foothold in the neuroscience and psychiatry fields, as it brings to market the first new schizophrenia drug in decades. It also validates the company’s $14 billion acquisition of Karuna, with execs predicting peak sales in the multibillion-dollar range. Researchers hope to broaden the use of Cobenfy in at least half a dozen other indications. — Max Gelman Active ingredient: inavolisib Indication: For adults with endocrine-resistant, PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy Snapshot : Roche’s Itovebi, which the company says is the first targeted therapy approved for people with HR-positive disease, got the green light from the FDA in October for patients with PIK3CA-mutated HR-positive HER2-negative breast cancer in combo with chemotherapy and Pfizer’s Ibrance. Patients on the drug saw progression-free survival of 15 months and a 58% overall response rate. — Katherine Lewin Active ingredient: marstacimab-hncq Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years and older with: hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors Snapshot : Pfizer had a few successes in hemophilia in 2024, first with a gene therapy approval in April and then the nod for Hympavzi. The drug reduces a naturally occurring anticoagulation protein, unlike other treatments, which replace a clotting factor. But the results over routine prophylaxis were “modest,” TD Cowen analysts have said. Pfizer will likely have to rely on its packaging as a pre-filled, auto-injector pen to get traction in the market, the analysts said. — Kyle LaHucik Active ingredient: zolbetuximab Indication: In combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors are Claudin18.2-positive, as determined by an FDA-approved test Snapshot : Vyloy, which targets a protein called Claudin18.2, scored an approval in combination with chemotherapy for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma. The drug was approved in Europe and Japan as well, making it the first in its class to get worldwide approval. — Katherine Lewin Active ingredient: sulopenem etzadroxil and probenecid Indication: Uncomplicated urinary tract infections caused by the designated microorganisms Escherichia coli , Klebsiella pneumoniae , or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options Snapshot : Iterum’s oral sulopenem was approved in October for certain patients with uncomplicated urinary tract infections (uUTIs). The drug, to be marketed as Orlynvah, is indicated specifically for patients who have limited or no alternative oral options. While the company had applied for broad use in adults with uUTIs, Iterum CEO Corey Fishman said the FDA’s label restrictions are “very consistent with how we’ve been thinking the whole time.” The agency had previously raised concerns that “inappropriate and/or extensive use” of an oral penem could result in cross-resistance with carbapenems, a class of antibiotics used for severe infections. — Nicole DeFeudis Active ingredient: obecabtagene autoleucel Indication: Adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia Snapshot : Autolus is challenging one of Gilead’s CAR-T cell therapies, Tecartus, with its recently approved treatment Aucatzyl in adult B-ALL. Aucatzyl will be listed at $525,000, making it pricier than Tecartus, but Autolus is hoping that its therapy’s safety profile will win over payers and providers. — Lei Lei Wu Active ingredient: eladocagene exuparvovec-tneq Indication: Adult and pediatric patients with aromatic 13 L-amino acid decarboxylase deficiency Snapshot : Known as Upstaza in Europe and Israel, PTC Therapeutics’ rare disease gene therapy finally won approval in the US in November under the brand name Kebilidi. The gene therapy is for AADC deficiency, an ultra-rare disease that impacts the nerve cells and brain. In the US, the therapy’s approval included children and adults across “the full spectrum of disease severity,” whereas the treatment’s previous approvals outside the country included patients with severe AADC deficiency. — Lei Lei Wu Active ingredient: revumenib Indication: Relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients 1 year and older Snapshot : Revuforj, also known as revumenib, is the first drug approved in a new class of medicines called menin inhibitors. It’s used for an aggressive form of leukemia driven by rearrangement of the KMT2A gene. However, it carries a boxed warning for differentiation syndrome, a potentially life-threatening reaction to some blood cancer treatments. Syndax plans to seek approval in 2025 for leukemia patients with a different and more common genetic alteration called NPM1. — Nicole DeFeudis Active ingredient: zanidatamab Indication: Adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer (BTC), as detected by an FDA-approved test Snapshot : Jazz Pharmaceuticals’ HER2-targeted bispecific antibody zanidatamab will be the first of its class to hit the market for biliary tract cancer. The drug, to be marketed as Ziihera, won accelerated approval in November for previously treated patients who scored high on an immunohistochemistry test measuring HER2 expression. A Phase 3 confirmatory trial is underway in the first-line setting, which executives say could support both a full approval in the second-line setting and a supplemental application for frontline patients. — Nicole DeFeudis Active ingredient: acoramidis Indication: Cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization Snapshot : Attruby, approved in November, is expected to compete with Pfizer’s Vyndaqel franchise and Alnylam’s Amvuttra, should the FDA greenlight the latter ahead of its March 23 decision date. BridgeBio and Alnylam also traded blows as the drugs moved through the clinic (Vyndaqel was approved in 2019). Notably, Attruby’s label said the drug reduces the likelihood of both hospitalization and death, putting it on the same ground as Vyndaqel. — Max Gelman Active ingredient: zenocutuzumab-zbco Indication: Pancreatic adenocarcinoma or NSCLC that harbors NRG1 gene fusions and are advanced unresectable or metastatic Snapshot : The new cancer drug marked the first commercial product for Merus. But it will be Partner Therapeutics that actually commercializes the product in the US, after paying for rights in 2024. — Max Bayer Active ingredient: cosibelimab-ipdl Indication: Adults with locally advanced or metastatic cutaneous squamous cell carcinoma who cannot receive curative surgery or radiation Snapshot : A new PD-L1 drug in 2024? Apparently so. Checkpoint Therapeutics won approval late in the year for its checkpoint inhibitor cosibelimab, which will be branded as Unloxcyt. The drug won approval for a form of advanced skin cancer, where Merck’s Keytruda and Regeneron’s Libtayo are already approved. Checkpoint was part of an earlier wave of companies that wanted to disrupt how the blockbuster class of cancer drugs are priced by selling their treatments at a discount compared to currently approved options, but those aspirations have failed to become reality. Checkpoint now says it believes its drug can be priced “at or near parity with other approved anti-PD-(L)1 therapies,” and the company is seeking a partner or business deal before launching the drug commercially. — Lei Lei Wu Active ingredient: crinecerfont Indication: To be used together with glucocorticoids to control androgen levels in adults and pediatric patients 4 years and older with classic congenital adrenal hyperplasia Snapshot : Neurocrine’s second approved product marked the first new drug for congenital adrenal hyperplasia in 70 years. It also brings some of the work of the company’s late founder, Wylie Vale, to fruition: Vale, who died in 2012, identified the compound that this drug works to block. Analysts at William Blair believe the drug could reach nearly $1.5 billion in peak sales by 2030. — Max Bayer Active ingredient: ensartinib Indication: Adult patients with anaplastic lymphoma kinase-positive locally advanced or metastatic non-small cell lung cancer who have not previously received an ALK inhibitor Snapshot : Xcovery won its first-ever FDA approval for a first-line treatment of adults with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer. The approval was based on a trial of 290 patients who had not previously received an ALK-targeted therapy, and FDA said Ensacove demonstrated a statistically significant progression-free survival improvement compared to Pfizer’s Xalkori. Xcovery is also testing Ensacove in other cancers. — Zachary Brennan Active ingredient: remestemcel-L-rknd Indication: Steroid-refractory acute graft-versus-host disease in pediatric patients 2 months and older Snapshot : Ryoncil took a winding path to approval, including two rejections and concerns from regulators about the design of its single-arm trial. An approval came more than four years after regulators accepted Mesoblast’s first application. But CEO Silviu Itescu believes it was worth the wait. Ryoncil is now the first mesenchymal stromal cell therapy to be approved by the FDA. It will be available for pediatric patients 2 months and older with acute graft-versus-host disease, a complication of allogeneic transplant in which donor cells attack the recipient. — Nicole DeFeudis Active ingredient: olezarsen Indication: As an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome Snapshot : Ionis’ antisense oligonucleotide olezarsen is the first approved treatment for familial chylomicronemia syndrome, a rare metabolic disorder that prevents the body from breaking down fats called triglycerides. The approval marks an important transition for Ionis, as Tryngolza will be the company’s first independent launch following years of partnerships. While FCS affects few people in the US, CEO Brett Monia hopes that a potential label expansion to a broader condition called severe hypertriglyceridemia could make Tryngolza a blockbuster. — Nicole DeFeudis Active ingredient: concizumab-mtci Indication: For routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B with inhibitors Snapshot : The anti-tissue factor pathway inhibitor Alhemo finally came good for Novo Nordisk at the tail end of 2024 following its rejection by the FDA in May 2023. It is the first daily subcutaneous treatment for hemophilia A or B patients with inhibitors — antibodies that neutralize clotting factors — to be delivered via a pen injector. It will compete with Pfizer’s similar weekly shot Hympavzi, but could reach more patients since Hympavzi is only approved for hemophilia A and B patients without inhibitors. — Elizabeth Cairns Active ingredient: vanzacaftor, tezacaftor, and deutivacaftor Indication: Cystic fibrosis in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the CFTR gene Snapshot : Alyftrek is Vertex’s “vanza triple” combination treatment for cystic fibrosis, a modulator of the CFTR gene. It’s the fifth such therapy Vertex has pushed across the finish line, but it comes with a boxed warning for potential drug-induced liver injury and failure. The treatment succeeded in its Phase 3 trials earlier this year, and Vertex used a priority review voucher to reduce the FDA’s review time to six months. — Max Gelman