Vadadustat

New Commercial Supply Contracts for Vafseo® (vadadustat) secured, bringing availability and dialysis organization coverage to nearly 100% of patients on dialysis in U.S. Vafseo tablets now shipping in the U.S. Plan to start Phase 3 trial in mid-2025 to potentially expand Vafseo label to include treatment of late-stage non-dialysis CKD patients Company to discuss business updates at 43rd Annual J.P. Morgan Healthcare Conference CAMBRIDGE, Mass., Jan. 13, 2025 /PRNewswire/ -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced multiple positive business updates. As of January, Vafseo® (vadadustat) tablets are available in the U.S. for adult patients with anemia due to chronic kidney disease (CKD) on dialysis for at least three months, and shipment of product to dialysis centers has commenced. In addition, Akebia recently signed a Vafseo commercial supply agreement with a leading dialysis organization. Akebia now has contracts in place with dialysis organizations caring for nearly 100% of dialysis patients in the U.S. As previously announced, Vafseo qualifies for the Transitional Drug Add-On Payment Adjustment (TDAPA) reimbursement as directed by the Centers for Medicare & Medicaid Services (CMS), and CMS has published billing guidance and reimbursement rates. Akebia also announced its plans to begin a Phase 3 trial for the use of vadadustat in treating anemia in late-stage CKD patients who are not on dialysis. Akebia received feedback from the U.S. Food and Drug Administration (FDA) on its protocol submission for a label expansion study and is incorporating feedback as appropriate. Akebia expects to begin the trial in mid-2025. "We believe Akebia is entering a transformational year with the U.S. market availability of Vafseo, which we believe could become a new standard of care for patients with CKD," said John P. Butler, Chief Executive Officer of Akebia. "During 2025 our commercial organization will remain focused on executing the U.S. launch of Vafseo, while our development team pursues label expansion of Vafseo into the non-dialysis population, which represents a potential multiple billion-dollar market opportunity in the U.S. We believe our commercial products and pipeline have the potential to generate significant shareholder value as Akebia strengthens its leadership position in the treatment of kidney disease and the hypoxia-inducible factor (HIF) space." Vafseo Commercial Update: Vafseo began shipping to dialysis centers and authorized distributors on January 9, 2025. Akebia has secured broad access for Vafseo as the company has now entered into commercial supply agreements with dialysis organizations treating nearly 100% of patients on dialysis in the U.S., including recently signing agreements with a leading dialysis organization and a mid-size dialysis provider. Akebia market research suggests 99% of nephrologists would consider prescribing Vafseo to certain eligible patients, with 75% intending to do so by 6 months after product availability. In December 2024, Akebia announced that U.S. Renal Care enrolled the first patients in the VOICE collaborative clinical trial of Vafseo. U.S. Renal Care has now enrolled more than 650 patients in the trial. Planned Phase 3 Trial of Vadadustat in U.S. Non-Dialysis CKD Patients: In March 2024, the FDA approved Vafseo for the treatment of anemia due to chronic kidney disease in adults who have been receiving dialysis for at least three months. The FDA acknowledged an unmet need for safer and orally available therapies to treat anemia due to CKD in certain non-dialysis patients. Akebia has since engaged with the FDA on potentially expanding vadadustat treatment to this patient population and has received feedback on a protocol submitted for a Phase 3 cardiovascular outcome study of approximately 1,500 U.S. subjects with late-stage CKD anemia not on dialysis, which is expected to begin in mid-2025. Akebia plans to request a Type C meeting with the FDA to continue to discuss the statistical analysis plan and regulatory path. Akebia expects its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including the U.S. Vafseo launch and planned pipeline advancement, for at least two years. Presentation at 43rd J.P. Morgan Healthcare Conference John Butler, Chief Executive Officer, will present on Thursday, January 16th, 2025 at 7:30 a.m. PST. A webcast of the presentation can be accessed through the "Investors" section of Akebia's website for 30 days following the conference. About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at , which does not form a part of this release. About Vafseo® (vadadustat) tablets Vafseo® (vadadustat) tablets is a once-daily oral hypoxia-inducible factor prolyl hydroxylase inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin, increasing hemoglobin and red blood cell production to manage anemia. Vafseo is approved for use in 37 countries. INDICATION VAFSEO is indicated for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Limitations of Use VAFSEO has not been shown to improve quality of life, fatigue, or patient well-being. VAFSEO is not indicated for use: As a substitute for red blood cell transfusions in patients who require immediate correction of anemia. In patients with anemia due to CKD not on dialysis. IMPORTANT SAFETY INFORMATION about VAFSEO (vadadustat) tablets CONTRAINDICATIONS Known hypersensitivity to VAFSEO or any of its components Uncontrolled hypertension WARNINGS AND PRECAUTIONS Increased Risk of Death, Myocardial Infarction (MI), Stroke, Venous Thromboembolism, and Thrombosis of Vascular Access A rise in hemoglobin (Hb) levels greater than 1 g/dL over 2 weeks can increase these risks. Avoid in patients with a history of MI, cerebrovascular event, or acute coronary syndrome within the 3 months prior to starting VAFSEO. Targeting a Hb level of greater than 11 g/dL is expected to further increase the risk of death and arterial and venous thrombotic events. Use the lowest effective dose to reduce the need for red blood cell (RBC) transfusions. Adhere to dosing and Hb monitoring recommendations to avoid excessive erythropoiesis. Hepatotoxicity Hepatocellular injury attributed to VAFSEO was reported in less than 1% of patients, including one severe case with jaundice. Elevated serum ALT, AST, and bilirubin levels were observed in 1.8%, 1.8%, and 0.3% of CKD patients treated with VAFSEO, respectively. Measure ALT, AST, and bilirubin before treatment and monthly for the first 6 months, then as clinically indicated. Discontinue VAFSEO if ALT or AST is persistently elevated or accompanied by elevated bilirubin. Not recommended in patients with cirrhosis or active, acute liver disease. Hypertension Worsening of hypertension was reported in 14% of VAFSEO and 17% of darbepoetin alfa patients. Serious worsening of hypertension was reported in 2.7% of VAFSEO and 3% of darbepoetin alfa patients. Cases of hypertensive crisis, including hypertensive encephalopathy and seizures, have also been reported in patients receiving VAFSEO. Monitor blood pressure. Adjust anti-hypertensive therapy as needed. Seizures Seizures occurred in 1.6% of VAFSEO and 1.6% of darbepoetin alfa patients. Monitor for new-onset seizures, premonitory symptoms, or change in seizure frequency. Gastrointestinal (GI) Erosion Gastric or esophageal erosions occurred in 6.4% of VAFSEO and 5.3% of darbepoetin alfa patients. Serious GI erosions, including GI bleeding and the need for RBC transfusions, were reported in 3.4% of VAFSEO and 3.3% of darbepoetin alfa patients. Consider this risk in patients at increased risk of GI erosion. Advise patients about signs of erosions and GI bleeding and urge them to seek prompt medical care if present. Serious Adverse Reactions in Patients with Anemia Due to CKD and Not on Dialysis The safety of VAFSEO has not been established for the treatment of anemia due to CKD in adults not on dialysis and its use is not recommended in this setting. In large clinical trials in adults with anemia of CKD who were not on dialysis, an increased risk of mortality, stroke, MI, serious acute kidney injury, serious hepatic injury, and serious GI erosions was observed in patients treated with VAFSEO compared to darbepoetin alfa. Malignancy VAFSEO has not been studied and is not recommended in patients with active malignancies. Malignancies were observed in 2.2% of VAFSEO and 3.0% of darbepoetin alfa patients. No evidence of increased carcinogenicity was observed in animal studies. ADVERSE REACTIONS The most common adverse reactions (occurring at ≥ 10%) were hypertension and diarrhea. DRUG INTERACTIONS Iron supplements and iron-containing phosphate binders: Administer VAFSEO at least 1 hour before products containing iron. Non-iron-containing phosphate binders: Administer VAFSEO at least 1 hour before or 2 hours after non-iron-containing phosphate binders. BCRP substrates: Monitor for signs of substrate adverse reactions and consider dose reduction. Statins: Monitor for statin-related adverse reactions. Limit the daily dose of simvastatin to 20 mg and rosuvastatin to 5 mg. USE IN SPECIFIC POPULATIONS Pregnancy: May cause fetal harm. Lactation: Breastfeeding not recommended until two days after the final dose. Hepatic Impairment : Not recommended in patients with cirrhosis or active, acute liver disease. Please note that this information is not comprehensive. Please click here for the Full Prescribing Information, including BOXED WARNING and Medication Guide. Forward-Looking Statements Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia's plans with respect to its U.S. commercial launch of Vafseo, including Akebia's statements regarding commercial supply contract coverage for Vafseo with dialysis organizations and Akebia's ability to make Vafseo available to nearly 100% of patients on dialysis in the U.S.; Akebia's plans and expectations with respect to potential Vafseo label expansion to treat anemia in late-stage CKD patients who are not on dialysis, including Akebia's expectations as to the timing of a Phase 3 trial, Akebia's statements regarding feedback from the FDA on its protocol submission for a label expansion study and Akebia's plans to request a Type C meeting with the FDA to discuss the statistical plan and regulatory path; Akebia's belief that it is entering a transformational year with U.S. market availability of Vafseo, and Akebia's belief that Vafseo could become a new standard of care for patients with CKD; Akebia's expectations regarding label expansion of Vafseo into the non-dialysis population, including a potential multiple billion-dollar market opportunity in the U.S.; Akebia's belief that its commercial products and pipeline have the potential to generate significant shareholder value as Akebia strengthens its leadership position in the treatment of kidney disease and the HIF space; Akebia's expectations regarding the demand for Vafseo from prescribers; and Akebia's expectations that its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including the U.S. Vafseo launch and planned pipeline advancement, for at least two years. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: the commercial availability of Vafseo; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Auryxia® and Vafseo, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia and Vafseo, including potential generic entrants; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to implement cost avoidance measures and reduce operating expenses; decisions made by health authorities, such as the FDA, with respect to regulatory filings and other interactions; the potential therapeutic benefits, safety profile, and effectiveness of Vafseo; the results of preclinical and clinical research; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; and early termination of any of Akebia's collaborations. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Akebia Therapeutics®, Auryxia and Vafseo® are registered trademarks of Akebia Therapeutics, Inc. and its affiliates. Akebia Therapeutics Contact Mercedes Carrasco [email protected] SOURCE Akebia Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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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The under-the-skin drug, when combined with background PAH therapy, helped patients walk farther than patients on placebo and background treatment in a Phase 3 trial. “The increase in six-minute walk distance capability was gargantuan,” Eliav Barr, chief medical officer at Merck Research Laboratories, said in an interview with Endpoints News . — Kyle LaHucik Active ingredient: vadadustat Indication: Anemia from chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months Snapshot : After securing approval in more than 35 other countries, Akebia’s anemia drug finally got the FDA’s blessing in March, almost two years to the day after it was initially rejected by the agency. The once-daily Vafseo tablets were greenlit for adults who have been on dialysis for at least three months and indicated for anemia due to chronic kidney disease. The drug belongs to a class of medicines known as hypoxia-inducible factor prolyl hydroxylase inhibitors. 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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