FDA Approves New Therapies for Hemophilia, Colorectal Cancer, and Cystic Fibrosis
Key Insights
The FDA approved Alhemo (concizumab) from Novo Nordisk for hemophilia A (search) and B with inhibitors, offering a once-daily subcutaneous option.
Pfizer's Braftovi (encorafenib) received accelerated approval as a first-line treatment for BRAF V600E (search)-mutated metastatic colorectal cancer (search) in combination with other therapies.
Vertex Pharma's Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a new triplet therapy for cystic fibrosis (search), has been approved for patients aged six and older with responsive mutations.
The FDA has recently approved several new drugs, providing advancements in the treatment of hemophilia, colorectal cancer (search), and cystic fibrosis (search). These approvals include Novo Nordisk's Alhemo (concizumab) for hemophilia, Pfizer's Braftovi (encorafenib) for colorectal cancer, Vertex's Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) for cystic fibrosis, and Hikma (search)'s generic version of Novo Nordisk's Victoza (liraglutide) for type 2 diabetes (search). These approvals address unmet medical needs and offer new treatment options for patients.
Alhemo Approved for Hemophilia A and B with Inhibitors
Novo Nordisk's Alhemo (concizumab), a tissue factor pathway inhibitor (search) (TFPI) antagonist, has been approved for the prevention or reduction of bleeding episodes in patients aged 12 years and older with hemophilia A (search) or B with inhibitors. Administered as a once-daily subcutaneous injection, Alhemo offers an alternative to existing treatments, particularly for patients who have developed inhibitors, which can reduce the effectiveness of clotting factor replacement therapy.
The approval is based on the phase 3 explorer study, which demonstrated an 86% reduction in treated spontaneous and traumatic bleeds compared to no prophylaxis. Amy Shapiro, co-medical director of the Indiana Hemophilia & Thrombosis Center, noted that the development of inhibitors remains the most serious treatment-related complication for people living with hemophilia, especially in hemophilia B (search) where the condition may remain poorly controlled and pose a life-threatening risk. Alhemo is the second drug in the TFPI class to be approved by the FDA, following Pfizer's Hympavzi (marstacimab). Both will compete with Roche's Hemlibra (emicizumab), a bispecific antibody targeting Factor IXa and factor X.
Braftovi Gains First-Line Approval for BRAF V600E-Mutated Metastatic Colorectal Cancer
Pfizer's BRAF inhibitor Braftovi (encorafenib) has received accelerated approval from the FDA as a first-line therapy for BRAF V600E (search)-mutated metastatic colorectal cancer (search). This approval marks a significant advancement, as no approved biomarker-driven therapies were previously specifically indicated for this patient population. The drug is approved for use in combination with Eli Lilly's EGFR inhibitor Erbitux (cetuximab) and chemotherapy.
The approval stems from the BREAKWATER trial, which showed an objective response rate (ORR) of 61% and a median duration of response of nearly 14 months, compared to an ORR of 40% and a duration of response of 11.1 months in the control group receiving chemotherapy with or without bevacizumab. BRAF mutations occur in approximately 10% of colorectal cancers. This is the fourth FDA-approved indication for Braftovi, potentially driving sales above $1 billion by 2028. Pfizer reported $213 million in sales from the product in 2023.
Alyftrek Approved as New Triplet Therapy for Cystic Fibrosis
Vertex Pharma has received FDA approval for Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a new triple therapy for cystic fibrosis (search) (CF). Alyftrek is designed to replace Vertex's top-selling Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor), which generated over $7.5 billion in global sales in the first nine months of the year. Alyftrek is approved for patients aged six years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies, potentially making another 150 people in the US eligible for treatment for the first time. A key advantage is its once-daily dosing, compared to Trikafta's twice-daily regimen.
Data from the SKYLINE 102 and SKYLINE 103 trials demonstrated that Alyftrek matched Trikafta in maintaining lung function in CF patients and was superior in reducing levels of chloride in sweat, a biomarker for disease control. Vertex has priced Alyftrek at $370,269 per year, 7% higher than Trikafta's wholesale acquisition cost (WAC) of $346,048 per year.
First Generic Liraglutide Approved for Type 2 Diabetes
The FDA has approved the first generic version of Novo Nordisk's once-daily injectable GLP-1 agonist Victoza (liraglutide), used to treat type 2 diabetes (search), from Hikma (search) Pharma. Victoza has been largely superseded by Novo Nordisk's once-weekly therapy Ozempic (semaglutide), with sales falling 41% in the first nine months of this year to $571 million as Ozempic rose by a third to $12 billion. The FDA prioritizes the review of generic versions of drugs that are in short supply, and liraglutide has been on the shortages list since 2023.