FDA Gears Up for Critical Approval Decisions in Q4 2024, Shaping Future Treatment Landscape
Key Insights
The FDA is expected to decide on BridgeBio's acoramidis for transthyretin amyloidosis cardiomyopathy (search) by November 29, potentially offering a new treatment option.
AstraZeneca and Daiichi Sankyo await a decision by December 20 on datopotamab deruxtecan for non-small cell lung cancer (search), aiming to supplant chemotherapy.
Vertex anticipates a decision by January 2, 2025, on its 'vanza triple' combination therapy for cystic fibrosis (search), offering improved convenience and patent protection.
The fourth quarter of 2024 is set to be pivotal for several pharmaceutical companies awaiting key FDA approval decisions that could reshape treatment paradigms across multiple disease areas. These decisions span from rare diseases like transthyretin amyloidosis cardiomyopathy (search) (ATTR-CM) and AADC deficiency (search) to more prevalent conditions like lung cancer and cystic fibrosis (search).
Acoramidis for Transthyretin Amyloidosis Cardiomyopathy
BridgeBio Pharma is anticipating a decision on acoramidis for ATTR-CM, a rare and often underdiagnosed condition affecting the heart. The FDA's decision is expected by November 29. Acoramidis, like Pfizer's Vyndamax, aims to stabilize the transthyretin protein, preventing misfolding and subsequent amyloid deposition in the heart. While preclinical data suggested acoramidis might offer superior efficacy, clinical trial results have been complex. In a Phase 3 trial, acoramidis initially missed its primary endpoint related to walking distance, but later demonstrated positive outcomes in overall survival and reduced hospitalizations. If approved, acoramidis would join Vyndamax in the U.S. market, potentially creating complex treatment decisions for physicians as the comparative effectiveness of the two drugs remains unclear.
Datopotamab Deruxtecan for Lung Cancer
AstraZeneca and Daiichi Sankyo are awaiting a decision by December 20 on datopotamab deruxtecan (dato-dxd) for non-small cell lung cancer (search) (NSCLC). This antibody-drug conjugate (ADC) targets the TROP2 (search) protein expressed in NSCLC tumors. Initial trials aimed for broad approval across NSCLC patients expressing TROP2. However, while dato-dxd demonstrated delayed tumor progression compared to chemotherapy, it did not significantly extend overall survival in the broader population. Subsequent analyses focused on patients with non-squamous NSCLC, revealing a numerical, though not statistically significant, improvement in survival. The companies are now seeking approval specifically for non-squamous NSCLC. The FDA's decision hinges on whether the observed benefits in this subpopulation are sufficient for approval, with some analysts suggesting a potential delay and an advisory committee meeting.
'Vanza Triple' for Cystic Fibrosis
Vertex Pharmaceuticals could further solidify its dominance in cystic fibrosis (search) (CF) treatment with the potential approval of its next-generation combination therapy, the 'vanza triple'. The FDA's decision is anticipated by January 2, 2025. The 'vanza triple' builds upon the foundation of Vertex's current blockbuster, Trikafta, utilizing tezacaftor as a backbone but incorporating newer compounds to enhance efficacy. Clinical trials have demonstrated that the 'vanza triple' is non-inferior to Trikafta in terms of lung function (ppFEV1) and superior in sweat chloride reduction, a key indicator of CFTR protein function. If approved, the 'vanza triple' offers the advantage of once-daily dosing compared to Trikafta's twice-daily regimen, along with extended patent protection and reduced royalty obligations for Vertex.
Upstaza for AADC Deficiency
PTC Therapeutics is seeking FDA approval for Upstaza, a gene therapy for AADC deficiency (search), a rare genetic nervous system disorder. The FDA decision is expected by November 13. Upstaza has already been approved in Europe, but the U.S. regulatory process has been prolonged due to data comparability issues. Approval would add Upstaza to the growing list of approved gene therapies for inherited diseases, though commercial success in this space remains challenging.
Subcutaneous Opdivo for Solid Tumors
Bristol Myers Squibb (search) awaits a decision by December 29 on a subcutaneous formulation of its blockbuster immunotherapy, Opdivo (nivolumab). This new formulation, developed in partnership with Halozyme, utilizes Halozyme's ENHANZE technology to enable under-the-skin injection, offering a more convenient administration route compared to the traditional intravenous infusion. Clinical trials have demonstrated that subcutaneous Opdivo is non-inferior to intravenous Opdivo in terms of efficacy. Approval would allow Bristol Myers to extend Opdivo's market exclusivity and maintain revenue streams ahead of patent expirations and biosimilar competition.