Allergan and Molecular Partners announced that the FDA has accepted a biologics license application (BLA) and the European Medicines Agency (EMA) has validated a marketing authorization application (MAA) for Abicipar pegol, a novel, investigational DARPin therapy in patients with wet age-related macular degeneration (AMD). The FDA is expected to take action on the BLA mid-2020. A decision from the European Commission is expected in the second half of 2020.
The BLA and MAA filings are based on data from two phase 3 trials, CEDAR and SEQUOIA, which supported the noninferior efficacy of the Abicipar quarterly dosing regimen to maintain vision gains with more than 50 percent fewer injections versus ranibizumab (13 vs. 6) dosed monthly in the first year.
“Acceptance of our marketing applications brings us one step closer to offering physicians and patients a new treatment option that has the potential to reduce patient visits and injections while achieving and maintaining vision gains with quarterly dosing,” David Nicholson, Chief Research and Development Officer, Allergan, said in a company news release. “Today’s announcement reinforces Allergan’s continued commitment to eye care innovation and means patients are one step closer to receiving what we believe to be transformative treatment that will help address unmet needs for neovascular AMD patients.”
The identical, global phase 3 head-to-head pivotal trials, CEDAR and SEQUOIA, assessed the efficacy and safety of Abicipar compared with ranibizumab in treatment-naïve patients with neovascular AMD. The primary endpoint measured the proportion of treated patients with stable vision at week 52 and, in both studies, Abicipar demonstrated similar efficacy after 6 or 8 injections, compared to 13 ranibizumab injections in the first year of this study. The overall adverse events were similar among the three treatment arms (Abicipar dosed every 8 weeks, Abicipar dosed every 12 weeks, or ranibizumab dosed monthly).
“The FDA filing acceptance marks an important milestone for the DARPin technology as Abicipar becomes our first DARPin candidate to receive filing acceptance by the FDA,” Michael T. Stumpp, COO of Molecular Partners, said in the news release. “We’re excited for the potential Abicipar holds to become a true quarterly dosed anti-VEGF treatment in patients with nAMD to provide vision gains and improved quality of life.”
DARPin molecules are derived from naturally occurring binding proteins that consist of repeat sequences with capping structures at each end of the protein. DARPin molecules have three key properties that have made them an important investigational class of binding protein for researchers: high binding affinity, low molecular weight and customizable applications. These three properties make DARPin molecules candidates for a broad range of therapeutic applications and are currently being investigated in therapeutic categories such as ophthalmology, oncology and immuno-oncology. Allergan and Molecular Partners are committed to advancing patient care through the development of molecules such as Abicipar.