Focal Points: A busy week for the FDA and clinical trial results
FDA issues CRL to Astellas' Supplemental New Drug Application for avacincaptad pegol intravitreal solution (IZERVAY)
Astellas Pharma announced the United States Food and Drug Administration ( FDA ) has issued a Complete Response Letter (CRL) for avacincaptad pegol intravitreal solution (IZERVAY). In the November 15 CRL, the FDA stated it cannot approve a supplemental New Drug Application in its present form. IZERVAY had an expected Prescription Drug User Fee Act (PDUFA) date of November 19.
In a press release, Astellas noted that the FDA comments in the CRL were not related to the safety, efficacy or risk of use for IZERVAY. The lack of approval is instead related to a statistical matter pertaining to proposed labelling language for the product.
The CRL was sent in response to a supplemental New Drug Application for IZERVAY, a treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), which sought to include positive 2 year data in the US Prescribing Information. This data was gathered from results of the GATHER2 Phase 3 clinical trial, which evaluated the safety and efficacy of monthly and every other month dosing.
FDA accepts NDA from Aldeyra Therapeutics for reproxalap for dry eye
The FDA has accepted the resubmitted new drug application (NDA) from Aldeyra Therapeutics for its first-in-class investigational candidate, topical ocular reproxalap for the treatment of signs and symptoms of dry eye disease. At the same time, Aldeyra is choosing to expand their option agreement with AbbVie, granting the manufacturer more cash to bring the drug to market.
Alkeus Pharmaceuticals receives FDA Rare Pediatric Disease and Fast Track designations for gildeuretinol as treatment for Stargardt disease
Alkeus Pharmaceuticals, Inc. announced that gildeuretinol (ALK-001), an investigational oral therapy, has received Rare Pediatric Disease and Fast Track designations from the FDA for the treatment of Stargardt disease.
Oral gildeuretinol acetate (ALK-001) is a new chemical entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. In preclinical studies, gildeuretinol decreased vitamin A dimerization down to the normal rate and prevented retinal degeneration and loss of visual function in animals with Stargardt disease.
Melt Pharmaceuticals reports positive Phase 3 topline efficacy results for MELT-300
Melt Pharmaceuticals announced positive topline results from its pivotal Phase 3 study evaluating the safety and efficacy of its lead product candidate, MELT-300, a non-IV, non-opioid tablet for procedural sedation during cataract surgery.
According to the company, based on a Special Protocol Assessment agreement with the FDA earlier this year, the study design and these positive results support the necessary objectives required for a regulatory submission.
Adverum Biotechnologies announces topline 52-week results from LUNA Phase 2 trial and 4-year OPTIC results
Laurent Fischer , MD, president and chief executive officer of Adverum Biotechnologies shared his thoughts in a press release:
"Both OPTIC 2E11 results and LUNA efficacy data at 52 weeks show maintenance of visual and anatomic endpoints with over 80% reduction in injection burden and greater than 50% injection freedom. These consistent results are bolstered by our OPTIC long-term data where we have demonstrated stable therapeutic aflibercept levels through 5 years. The data across both studies support a reliable long-term benefit and a predictable safety profile."
Ocugen announces positive preliminary data from OCU410 Phase 1 clinical trial for geographic atrophy
Ocugen announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation segment of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).
Key findings include: