The FDA is poised to make critical decisions on several therapies in the coming weeks, impacting treatment options for rosacea, non-small cell lung cancer (NSCLC), pancreatic cancer, aromatic L-amino acid decarboxylase (AADC) deficiency, and acute lymphoblastic leukemia (ALL). These decisions could introduce new treatment modalities and address unmet needs in these diverse patient populations.
Journey Medical's DFD-29 for Rosacea
Journey Medical Corporation's DFD-29 (minocycline hydrochloride modified release capsules) is under FDA review as a potential oral treatment for inflammatory lesions and erythema in adults with rosacea, with a decision expected by November 4. Rosacea, affecting approximately 16 million people in the U.S., manifests as red, pus-filled bumps and facial redness. The New Drug Application (NDA) is supported by two Phase III studies where DFD-29 demonstrated significant superiority over the current standard of care, 40-mg Oracea (doxycycline) capsules, in treatment success based on Investigator’s Global Assessment. DFD-29 also outperformed Oracea and placebo in reducing total inflammatory lesion count and erythema. If approved, DFD-29 would be the only oral medication to address both inflammatory lesions and erythema in rosacea.
Merus' Zenocutuzumab for NRG1 Fusion-Positive Cancers
Merus, a Netherlands-based company, is seeking approval for zenocutuzumab, an investigational bispecific antibody for NSCLC and pancreatic ductal adenocarcinoma (PDAC) patients with fusions of the NRG1 gene. The FDA granted Priority Review to the Biologics License Application (BLA), setting a decision date of November 6. NRG1 encodes for the HER3 ligand neuregulin, and fusions involving this gene can drive cancer development. Zenocutuzumab utilizes Merus’ Dock & Block technology to bind to HER2 and disrupt the interaction between the HER3 receptor and NRG1-fusion proteins, preventing cancer cell proliferation. Phase I/II eNRGy study data showed a 37% objective response rate in NSCLC patients and a 42% objective response rate in PDAC patients with NRG1 fusions.
PTC Therapeutics' Upstaza for AADC Deficiency
PTC Therapeutics awaits the FDA's decision on Upstaza (eladocagene exuparvovec), a gene therapy for pediatric patients (18 months and older) with aromatic L-amino acid decarboxylase (AADC) deficiency, with a decision expected by November 13. AADC deficiency, a rare genetic disorder affecting between 1 in 64,000 and 1 in 90,000 births in the U.S., results from mutations in the DDC gene, leading to insufficient production of dopamine and serotonin. Upstaza delivers a functional copy of the DDC gene directly into the brain via a minimally invasive procedure. The European Commission granted Marketing Authorization for Upstaza in July 2022, and the U.K.’s Medicines and Healthcare Products Regulatory Agency followed suit in November 2022.
Autolus Therapeutics' Obe-cel for Acute Lymphoblastic Leukemia
Autolus Therapeutics anticipates the FDA's verdict on obecabtagene autoleucel (obe-cel), an investigational CAR T therapy for adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), by November 16. The BLA is supported by data from the Phase II FELIX study, which demonstrated a 70% overall remission rate with promising expansion and persistence at a median follow-up of 6.4 months. Obe-cel was generally well-tolerated, with 3% of patients experiencing grade 3 or higher cytokine release syndrome and 8% experiencing grade 3 or higher immune effector cell-associated neurotoxicity syndrome. If approved, obe-cel would join the limited number of approved CAR T therapies for ALL.
