Rare and Orphan diseases approved w smaller pt trial numbersMarch 27, 2024 - As outlined in the attached link, Pfizer's termination of one of two phase 3 trials assessing a monoclonal antibody designed to treat sickle cell disease, and due to slow recruitment in the general population, doesn’t impact Pfizer's plans for a potential new drug approval from the FDA in 2026, according to a Pfizer spokesperson since their monoclonal antibody Inclacumab has received both orphan and rare pediatric disease tags from the federal agency for sickle cell disease.
Drugs targeting Orphan and Rare diseases are approved early and on small numbers of clinical trial patients. ONCY's pelareorep in the treatment of pancreatic and metastatic breast cancer indications is a candidate for Accelerated Approval in both indications that are considered to have unmet treatment needs.
Furthermore, pelareorep is a biologic that will be provided 13 years of market exclusivity from the date of FDA approval.
https://www.fiercebiotech.com/biotech/after-terminating-1-2-late-stage-sickle-cell-disease-trials-pfizer-still-expects-fda