FDA Gears Up for Busy November with Key Decisions on Novel Therapies
核心洞察
The FDA is set to decide on Journey Medical's DFD-29 for rosacea (搜索), potentially the first oral systemic therapy addressing both inflammatory lesions and erythema.
Merus' Zenocutuzumab, a targeted therapy for NRG1+ lung and pancreatic cancers, is under priority review, offering a potential improvement over current treatments.
Decisions are pending for PTC Therapeutics' Upstaza for AADC deficiency (搜索), Autolus Therapeutics' Obe-cel for B-cell acute lymphoblastic leukemia (搜索), and Applied Therapeutics' Govorestat for Classic Galactosemia (搜索).
As November approaches, the FDA is preparing to make critical decisions on several novel therapies, impacting treatment landscapes across various diseases. Here's a look at the key drugs awaiting potential approval.
Journey Medical's DFD-29 for Rosacea
Journey Medical Corp. (搜索) anticipates an FDA decision on DFD-29 by November 4, 2024, for treating inflammatory lesions and erythema of rosacea (搜索) in adults. Clinical trials demonstrated DFD-29's statistical superiority over Oracea 40 mg capsules and placebo in Investigator's Global Assessment treatment success and reduction in inflammatory lesion count. If approved, DFD-29 would be the only oral, systemic therapy to address both inflammatory lesions and erythema from rosacea.
Merus' Zenocutuzumab for NRG1+ Cancers
Merus N.V.'s lead drug candidate, Zenocutuzumab (Zeno), is under priority review for treating patients with neuregulin 1 fusion (搜索) (NRG1+) non-small cell lung and NRG1+ pancreatic cancer (搜索). NRG1 fusions are oncogenic drivers found in various tumor types. The FDA accepted the Biologics License Application for Zeno on May 6, 2024, and a decision is expected by November 6, 2024. Zenocutuzumab has the potential to be the first and only targeted therapy for patients with NRG1+ lung and pancreatic cancer.
Astellas Pharma's IZERVAY Label Update
Astellas Pharma awaits an FDA decision on November 19, 2024, regarding a supplemental New Drug Application for IZERVAY, seeking to include positive 2-year data for treating geographic atrophy (搜索) secondary to age-related macular degeneration. The sNDA is based on results from the GATHER2 phase III clinical trial, which evaluated the efficacy and safety of monthly (EM) and every other month (EOM) dosing of IZERVAY through year 2. Data demonstrated that IZERVAY continued to reduce the rate of geographic atrophy lesion growth versus sham.
PTC Therapeutics' Upstaza for AADC Deficiency
PTC Therapeutics' Upstaza, a gene therapy for treating AADC deficiency (搜索), is under FDA review, with a decision anticipated on November 13, 2024. Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder affecting the brain, characterized by poor muscle tone, delayed development, and involuntary eye movements. Upstaza is a one-time gene replacement therapy proposed for patients aged 18 months and older with a genetically confirmed diagnosis of AADC deficiency with a severe phenotype. Clinical trials demonstrated transformational neurological improvements.
Autolus Therapeutics' Obe-cel for B-ALL
The FDA decision on Autolus Therapeutics' Obe-cel, proposed for treating patients with relapsed/refractory adult B-cell acute lymphoblastic leukemia (搜索), is expected on November 16, 2024. Obe-cel, also known as Obecabtagene autoleucel, is a CD19 (搜索) CAR T cell investigational therapy designed to overcome limitations in clinical activity and safety compared to current CD19 CAR T cell therapies. In a pivotal study, 78% of patients treated with Obe-cel achieved a complete response (CR) or CR with incomplete hematological recovery (CRi).
Applied Therapeutics' Govorestat for Classic Galactosemia
Applied Therapeutics' Govorestat, proposed for treating Classic Galactosemia (搜索), awaits the FDA decision on November 28, 2024. Govorestat is a novel Aldose Reductase Inhibitor that crosses the blood-brain barrier. Galactosemia is a rare, genetic disorder where the body cannot break down the sugar galactose, leading to a buildup of the toxic metabolite galactitol. There is no known cure or approved treatment for Galactosemia.
BridgeBio Pharma's Acoramidis for ATTR-CM
The FDA decision on BridgeBio Pharma's Acoramidis, an investigational drug for treating transthyretin amyloid cardiomyopathy (搜索), is due on November 29, 2024. Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare but severe cause of restrictive cardiomyopathy caused by the accumulation of transthyretin fibrils in the myocardium. In the ATTRibute-CM phase III trial, Acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual cardiovascular-related hospitalization (CVH) frequency.
Jazz Pharmaceuticals' Zanidatamab for HER2+ Biliary Tract Cancer
The FDA decision on Zanidatamab, the human epidermal growth factor receptor 2 (HER2 (搜索))-targeted bispecific antibody, for treating previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (搜索), is due on November 29, 2024. Zanidatamab is being developed by Jazz Pharmaceuticals and BeiGene, Ltd. If approved, Zanidatamab would be the first HER2-targeted therapy specifically for patients with locally advanced or metastatic HER2+ BTC.