Adverum Biotechnologies Inc. (NASDAQ: ADVM) has announced that the US FDA has granted ADVM-062 Orphan Drug Designation. ADVM-062 is a proprietary gene therapy candidate the company is developing as a possible single intravitreal administration for blue cone monochromacy through the delivery of a functional OP1LW gene copy.
ADVM-062 builds on the company’s ophthalmology expertise
The company’s CEO and President Laurent Fischer said, “We are excited to introduce ADVM-062, which builds on our ophthalmology and gene therapy expertise to address the significant unmet medical needs of patients living with blue cone monochromacy. We are pleased the FDA has recognized the patient need by granting Orphan Drug Designation and we look forward to advancing ADVM-062 towards the clinic and generating important data.”
BCM affects 1 to 9 boys in 100,000 over the world. Mutations either in L or M opsin gene(s) produce this X-linked recessive genetic disorder, resulting in visual acuity loss, myopia photophobia, and infantile nystagmus, which can last into adulthood. As a result, people with BCM have difficulty with crucial areas of daily life like facial recognition, reading, learning, and daylight vision. Unfortunately, there is currently no treatment for BCM.
ODD makes Adverum eligible for tax credits for clinical studies and user fee waivers
The FDA grants orphan drug designation to innovative medicines and biologics that are designed for the effective and safe treatment, diagnosis, or prevention of rare disorders or diseases affecting fewer than 200,000 persons in the United States. Sponsors who develop treatments for rare diseases may be eligible for tax credits for clinical trial costs and prescription drug user charges exemptions due to the classification. Should Adverum Biotechnologies achieve FDA approval for BCM treatment with ADVM-062, the orphan drug classification may give the firm to a seven-year duration of commercial exclusivity in the US.
Expected milestones for ADVM-062 include a plan to present preclinical data that support proof of concept of ADVM-062 in the first half of 2022 and the intention to file an investigational New Drug Application by the end of this year.
