Forma Therapeutics, Inc.

With its ability to specifically inhibit the mutant IDH1 enzyme, TIBSOVO addresses an unmet need in personalized cancer treatment, offering new hope for patients with limited options. As clinical evidence continues to support its efficacy and safety, TIBSOVO is positioned for strong growth in the oncology market, especially with expanding indications and global market penetration. LAS VEGAS, April 8, 2025 /PRNewswire/ -- DelveInsight's " TIBSOVO Market Size, Forecast, and Market Insight Report" highlights the details around TIBSOVO, the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of TIBSOVO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Servier's TIBSOVO (ivosidenib) Overview TIBSOVO (ivosidenib) is a small molecule developed by Servier, acts as an isocitrate dehydrogenase-1 (IDH1) inhibitor. It is indicated for patients with a susceptible IDH1 mutation, which is identified through an FDA-approved test. Currently, TIBSOVO is approved for the treatment of newly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, relapsed or refractory myelodysplastic syndromes (MDS), and locally advanced or metastatic cholangiocarcinoma. According to Servier's pipeline, TIBSOVO is also being actively investigated for a range of additional indications, with key areas of exploration including solid tumors and hematological malignancies. Learn more about TIBSOVO projected market size for AML, cholangiocarcinoma, and MDS @ TIBSOVO Market Potential The oncology market has experienced significant growth in recent years, driven by advances in cancer research, the development of targeted therapies, and the increasing global prevalence of cancer. As the second-leading cause of death worldwide, cancer continues to pose a major public health challenge, creating a substantial demand for innovative treatments. The market is marked by a shift toward personalized medicine, where therapies are tailored to an individual's genetic profile, allowing for more effective and less toxic treatments. Immunotherapies, such as checkpoint inhibitors, CAR-T cell therapies, and oncolytic virus therapies, are at the forefront of these advancements, offering new hope to patients with previously untreatable cancers. Moreover, the oncology market is expanding due to the growing adoption of precision oncology and molecular diagnostics, which enable earlier and more accurate detection of cancer. This has led to increased investments in research and development, especially in areas like liquid biopsies, companion diagnostics, and next-generation sequencing. The demand for more effective and accessible cancer treatments is fueling innovation in both the pharmaceutical and biotechnology sectors, with numerous new drugs and therapies entering clinical trials and moving toward commercialization. As a result, the oncology market is poised to continue growing, with significant opportunities for breakthrough therapies in immuno-oncology, targeted treatments, and gene therapies. DelveInsight has expertise in the oncology market with an experienced team handling the oncology domain proficiently. DelveInsight has released a series of epidemiology-based market reports on Acute Myeloid Leukemia, Cholangiocarcinoma, and Myelodysplastic Syndrome. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Gain deeper insights into these diseases with a customized report tailored to your needs. Connect with us today—our experts are ready to assist you! Emerging Competitors of TIBSOVO TIBSOVO faces significant competition in the newly diagnosed AML market from several alternative treatments. Oral therapies like VENCLEXTA (venetoclax), which inhibits BCL-2, and DAURISMO (glasdegib), which targets the hedgehog pathway, present strong contenders. Additionally, emerging therapies such as Lisaftoclax (Ascentage Pharma), another BCL-2 inhibitor, and gilteritinib (Astellas), a kinase inhibitor, are expected to further challenge TIBSOVO in the newly diagnosed AML space. In the R/R AML market, TIBSOVO competes with several therapies, including XOSPATA (gilteritinib), IDHIFA (enasidenib), and others. Emerging therapies such as Ziftomenib (Kura Oncology) and RVU-120 (Ryvu Therapeutics) are also expected to compete with TIBSOVO upon their approval. Ziftomenib, a small molecule developed by Kura Oncology, is currently in Phase I/II trials. In the relapsed/refractory (R/R) MDS market, TIBSOVO faces competition from therapies such as RYTELO (Imetelstat), an IV injection that inhibits oligonucleotide telomerase, and REBLOZYL (Luspatercept), a subcutaneous injection, among other treatment options. In the metastatic cholangiocarcinoma market, TIBSOVO faces relatively less competition, as there are few approved therapies. One such competitor is PEMAZYRE (pemigatinib), a kinase inhibitor that was approved in the US in 2020 and in Europe in 2021. Whereas, Tyra Biosciences' TYRA-200, an FGFR1/2/3 inhibitor, is in phase I stage of development that might be a threat to TIBSOVO one approved. To know more about the number of competing drugs in development, visit @ TIBSOVO Market Positioning Compared to Other Drugs Key Developmental Activities of TIBSOVO In October 2023, Servier announced that the FDA has approved TIBSOVO (ivosidenib tablets) for the treatment of IDH1-mutated R/R MDS. In May 2023, Servier announced that the European Commission has approved TIBSOVO as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed AML with an isocitrate dehydrogenase-1 R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. In May 2022, Servier announced FDA approval of TIBSOVO (ivosidenib tablets) in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia. In August 2021, Servier announced FDA approval for TIBSOVO (ivosidenib tablets) for the treatment of adult patients with previously treated, locally advanced, or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test. TIBSOVO is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. In May 2019, Agios announced FDA approval of TIBSOVO (ivosidenib tablets) as monotherapy for newly diagnosed adult patients with IDH1 mutant acute myeloid leukemia not eligible for intensive chemotherapy. In July 2018, the FDA approved TIBSOVO (ivosidenib) for relapsed or refractory acute myeloid leukemia with an idh1 mutation. In February 2018, the FDA accepted a new drug application and granted priority review for ivosidenib in relapsed or refractory AML with an IDH1 mutation. In December 2017, Agios submitted an NDA to the FDA for ivosidenib for the treatment of patients with relapsed/refractory aml and an IDH1 mutation. TIBSOVO Patent Details TIBSOVO is a drug owned by Servier Pharmaceuticals. Currently, it is protected by 10 Orange book-listed patents filed from 2018 to 2023, out of which none have expired yet. It is also protected by ODE exclusivity for various indications. Discover how TIBSOVO is shaping the blood cancer treatment landscape @ TIBSOVO Side Effects TIBSOVO Market Dynamics In recent years, the therapeutic landscape has undergone significant changes, with promising approaches including immunotherapy, chemotherapy combined with targeted treatments, and the regulation of immune checkpoint-mediated signaling pathways. One such treatment, Ivosidenib, works by inducing the bone marrow to develop normal blood cells, helping to reduce the frequency of blood transfusions. Investment in the research and development of drugs has surged, contributing to a rich pipeline and forecasting promising opportunities in the treatment markets for AML and cholangiocarcinomas. Therapies like TIBSOVO have the potential to capture market attention due to their efficacy and innovation. As healthcare spending continues to rise globally, the pharmaceutical market is expected to expand, providing greater opportunities for market penetration by manufacturers and facilitating the development and distribution of new treatments. Despite progress in cancer treatment, significant gaps remain in understanding the full process that governs carcinogenesis and resistance to treatments, particularly in cancers like acute myeloid leukemia (AML). This highlights the need for more advanced research to address these challenges. Additionally, the limited biomarker testing for patients with the IDH1 mutation restricts the targeted patient pool, posing a challenge for therapies like TIBSOVO to achieve their expected market share. The increasing competition from emerging therapies, such as KEYTRUDA (pembrolizumab), IMFINZI (durvalumab), and the combination of Azacitidine + Venetoclax, further intensifies the pressure on TIBSOVO. Moreover, the difficulty in obtaining sufficient patient pools and diagnostic samples during disease recurrence presents significant hurdles to the effective development of translational research, potentially slowing progress in optimizing treatments. Dive deeper to get more insight into TIBSOVO's strengths & weaknesses relative to competitors @ TIBSOVO Market Drug Report Table of Contents Related Reports Acute Myeloid Leukemia Market Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Relapsed/Refractory Acute Myeloid Leukemia Market Relapsed/Refractory Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key R/R AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Myelodysplastic Syndrome Market Myelodysplastic Syndrome Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key MDS companies such as Bristol-Myers Squibb, Astex Pharmaceutical, Taiho Oncology, Fibrogen, AbbVie, Gilead Sciences, Novartis, Syros Pharmaceuticals, Takeda, Pfizer, Geron Corporation, Karyopharm Therapeutics, Antengene Corporation, BerGenBio ASA, Jazz Pharmaceuticals, Aprea Therapeutics, Sanofi, Medac, among others. Cholangiocarcinoma Market Cholangiocarcinoma Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key cholangiocarcinoma companies such as AstraZeneca, Decalth Systems, Basilea Pharmaceutica, Taiho Oncology, Eisai Pharmaceuticals, TransThera Sciences, Incyte Corporation, Roche, Agios Pharmaceuticals, Servier Pharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

To date, Novo Nordisk’s focus in rare disease has hinged on blood and endocrine disorders, with its efforts yielding a number of approved treatments. While Novo Nordisk has been operating in rare disease for some 45 years, the field has often taken a back seat to the Danish drugmaker’s bread-and-butter business developing treatments for diabetes and other common chronic diseases.Still, Novo is steadfast in its goal to eventually become a leader in the space, the company’s EVP of rare disease, Ludovic Helfgott, said in an interview with Fierce Pharma following the company’s 2025 international press briefing on Thursday. Overall, the near-term success of those ambitions will come down to a handful of new drug launches and a sickle cell disease (SCD) candidate that recently entered phase 3 testing.Plus, Novo will continue to seek external opportunities to refine its rare disease pedigree, Helfgott said.From 'back burner' to full throttle Novo Nordisk's rare disease ambitions first began to shift when Lars Fruergaard Jørgensen donned the mantle of CEO in 2017.“One of the intuitions that Lars had when he joined the CEO chair was to say, we have here something which is an uncut gem—something which is a rough diamond that we might want to polish, because at some point it might be very, very helpful,” Helfgott said of the company’s rare disease unit.When Helfgott joined Novo from AstraZeneca in 2019, he immediately set out on a mission to create “a biotech within Novo Nordisk," he explained.“There was a long endgame in being a leader in rare disease,” Helfgott added.While Novo has subsequently made strides building out capabilities in early research, development and its supply chain to embrace that newfound purpose—including recent designs on a $1.2 billion rare disease production plant in Denmark—there is still much work to be done, Helfgott said.“When I joined the company back in 2019, the R&D efforts [in rare disease] honestly had been a bit put on the back burner, and we had to reengage with research,” he explained. Over the past few years, Novo has relied on its dealmaking prowess to wade deeper into the space, but in order to be a discerning purchaser and partner, the company had to bolster its own R&D abilities too, Helfgott said.“The problem is that if you don’t have your internal quality research and development, you don’t do good [business development] because you can’t assess quality molecules, and you’re not really a credible partner,” he explained.In an example of how Novo’s rare disease outfit has since grown, Helfgott noted that the company’s early research team for the business has swelled from just eight people to around 90 since the executive’s arrival.Those efforts to bulk up in rare disease R&D, both internally and externally, have since led to some major rare disease deals for Novo in recent years, including the company’s $1.1 billion buyout of sickle cell disease-focused Forma Therapeutics in 2022 and its acquisition of 2seventy bio’s hemophilia A program in 2024.Building a path to the top To date, Novo’s focus in rare disease has hinged on blood and endocrine disorders, with those efforts yielding a number of approved hemophilia A and B treatments like Esperoct, NovoEight and Rebinyn, as well as drugs for growth hormone-related disorders such as Norditropin.More recently, Novo in December won a hard-fought FDA approval for its once-daily hemophilia injection Alhemo. The tissue factor pathway inhibitor (TFPI) antagonist, which is injected under the skin, is specifically approved to prevent or curb the frequency of bleeding episodes in patients 12 and older who have hemophilia A or B with certain factor inhibitors.Factor inhibitors are a complication of the rare bleeding disorder that can prevent factor replacement treatments—a well-established class of drugs for hemophilia—from working properly.Given Alhemo’s unique mode of action, much of Novo’s launch focus in the U.S. currently revolves around investments in medical education, Helfgott said. The company is also playing up the drug’s convenient subcutaneous delivery via prefilled syringe, which is a first in the hemophilia B space, where many medicines are infused, the executive pointed out.Outside the U.S., Alhemo has also won green lights in Australia, Japan and Switzerland and the European Union.Beyond hemophilia, the next frontier for Novo’s blood disorder focus is SCD, where the company recently advanced the asset etavopivat it picked up from Forma Therapeutics into phase 3. Patients with SCD are living with “two evils,” to hear Helfgott tell it. They’re forced to endure painful vaso-occlusive crises (VOCs), in which blood vessels are blocked and deprive tissues of oxygen, as well as the subsequent organ damage that results from those repeated VOCs, he explained.Novo hopes that etavopivat can tackle both issues at once, which even newer SCD meds have failed to achieve, Helfgott said.Back in December, Novo posted phase 2 data on etavopivat showing the highest studied dose (400 mg) nearly halved patients’ annualized VOC rates at the study’s 52-week mark compared with placebo. Etavopivat also appeared to delay the onset of patients’ first VOC, according to data presented at last year’s meeting of the American Society of Hematology (ASH).What's next for Novo in rare diseases As Novo looks to carve out a leadership position in the field, the company’s rare disease unit will retain its focus on blood and growth disorders, Helfgott said.“It’s our core, and we will continue to develop this both internally and externally,” he explained.As for how Novo aims to dig deeper into those disciplines, the company has grown “increasingly interested” in what it calls the “hemato-renal area,” which refers to the confluence between rare blood and kidney disorders, Helfgott said.To that end, Novo in late 2023 won an FDA nod for its ribonucleic acid interference (RNAi) drug Rivfloza to treat the rare disease primary hyperoxaluria type 1 (PH1). The disease is characterized by an overproduction of oxalate, which causes kidney stones to form and lead to progressive kidney damage.Rivfloza kicked off its U.S. launch “a few months ago,” with a European rollout soon to follow, Helfgott said. Meanwhile, M&A and other business development deals aren’t off the table as Novo looks to expand in blood, hemato-renal and endocrine disorders, the executive pointed out.External dealmaking could also deepen Novo’s access to new and promising modalities, as was the case with the $600 million collaboration agreement the company inked with NanoVation Therapeutics in September.Under that accord, Novo has secured a license to use NanoVation’s long-circulating lipid nanoparticle technology to develop two base-editing therapies in rare genetic diseases. The deal also covers up to five more targets in rare and cardiometabolic conditions. 

Agios’ blood disease treatment Pyrukynd passed a Phase 3 study in PK deficiency in children who do not regularly receive blood transfusions. The readout arrives after Pyrukynd last year failed to meet the primary endpoint in a separate Phase 3 study of children with PK deficiency who are regularly transfused. Pyruvate kinase deficiency is a rare genetic disease in which red blood cells are prematurely destroyed. In its announcement on Thursday, Agios said that Pyrukynd, also known as mitapivat, hit the primary endpoint of hemoglobin response in the ACTIVATE-Kids study, which enrolled 30 children aged 1 year or older who do not receive regular blood transfusions. The biotech said that six of 19 patients who received Pyrukynd saw a sustained increase (≥1.5 g/dL) in their hemoglobin levels by 20 weeks. Meanwhile, none of the 11 patients in the placebo arm reached the bar of hemoglobin response. Safety-wise, there was a similar proportion of patients who had adverse events in both study arms, and there were no discontinuations in the study, according to Agios. Back in December, the biotech disclosed that five patients in its Phase 3 thalassemia study experienced liver toxicity, two of which were considered serious adverse events. Notably, Agios now plans to ask for approval of Pyrukynd in children with PK deficiency based on the results of both Phase 3 studies, despite the fact that the previous trial in children who are regularly transfused missed its key endpoint. Pyrukynd is currently approved in the US to treat hemolytic anemia in adults with PK deficiency. Agios is also studying the therapy in other conditions like thalassemia and sickle cell disease. In its earnings update on Thursday, Agios said it has submitted Pyrukynd for approval in adults with thalassemia in the US, where the FDA has a decision deadline of Sept. 7. It’s seeking approval regardless of transfusion status and for both alpha- and beta-thalassemia patients. The biotech has also asked for approval for the same indication in the EU, Saudi Arabia and UAE. Pyrukynd is what’s known as a pyruvate kinase activator. Pyruvate kinase is a key enzyme in red blood cells that converts sugar into energy. The belief is that increasing its activity will keep red blood cells from breaking down prematurely. Novo Nordisk is developing a competing treatment called etavopivat, which it acquired as part of its $1.1 billion purchase of Forma Therapeutics in 2022. It’s studying etavopivat in sickle cell disease and thalassemia, and it announced in December that etavopivat is heading to pivotal trials in sickle cell disease.