While a significant number of clinical trial delays occurred during the month of April, it was business as usual from a regulatory standpoint for companies with late-stage therapies ready for the market and for those targeting underserved patient populations.
BioWorld tracked a total of 201 regulatory news items in April, as well as 22 clinical data reports coming out of the American Association for Cancer Research (AACR). Within the regulatory data, there were 45 entries – nearly a quarter – concerning global approvals for companies to begin testing of various therapeutics or vaccines for COVID-19 and its complications resulting from the SARS-CoV-2 infection.
The FDA granted fast track designations for 11 therapies in April, including two for pancreatic cancer candidates, Lyon, France-based Erytech Pharma SA’s Eryaspase and Hong Kong-based Hutchison China Meditech Ltd.’s surufatinib, which is also targeting non-pancreatic neuroendocrine tumors. Other fast track designations went to Lexington, Mass.-based Agenus Inc.’s anti-PD-1 antibody balstilimab for cervical cancer and to Morris Plains, N.J.-based Immunomedics Inc.’s sacituzumab govitecan for locally advanced or metastatic urothelial cancer. The latter drug was approved later in April as Trodelvy for metastatic triple-negative breast cancer patients who had received at least two prior therapies.
Trodelvy was one of five new molecular entities approved by the FDA during the month. Others included Koselugo (selumetinib) for neurofibromatosis type 1, from Cambridge, U.K.-based Astrazeneca plc and Kenilworth, N.J.-based Merck & Co. Inc.; Ongentys (opicapone) for Parkinson’s disease, from San Diego-based Neurocrine Biosciences Inc.; Pemazyre (pemigatinib) for locally advanced or metastatic cholangiocarcinoma, from Wilmington, Del.-based Incyte Corp.; and Tukysa (tucatinib) for breast cancer, from Bothell, Wash.-based Seattle Genetics Inc.
Other notable approvals in April are for Paris-based Sanofi SA’s Menquadfi for meningococcal disease, Princeton, N.J.-based Urogen Pharma Inc.’s Jelmyto (mitomycin gel) for low-grade upper tract urothelial cancer and High Point, N.C.-based Banner Life Sciences LLC’s Bafiertam (monomethyl fumarate) for relapsing multiple sclerosis.
A total of eight biopharma therapies received PDUFA dates during April, five of which have regulatory filings that are under priority review. Those five include Kenilworth, N.J.-based Merck & Co. Inc.’s Keytruda (pembrolizumab) to treat unresectable or metastatic solid tumors (June 16, 2020); New York-based Bristol Myers Squibb Co.’s Opdivo (nivolumab) and Yervoy (ipilimumab) as a first-line treatment of metastatic or recurrent non-small-cell lung cancer with no EGFR or ALK genomic tumor aberrations (Aug, 6, 2020); Research Triangle Park, N.C.-based Fennec Pharmaceuticals Inc.’s Pedmark, a formulation of sodium thiosulfate, to prevent ototoxicity induced by cisplatin chemotherapy in patients with localized, nonmetastatic solid tumors. (Aug. 10, 2020); Sydney-based Mesoblast Ltd.’s Ryoncil (remestemcel-L) for graft-vs.-host disease (Sept. 30, 2020); and Tarrytown, N.Y.-based Regeneron Pharmaceuticals Inc.’s REGN-EB3 for Ebola virus infection (Oct. 25, 2020).
April regulatory data, as well as clinical results presented through AACR, can be found at the following links:
Biopharma financings data for 2020 through May 14 can be found here: