May was a big month for developers of non-small-cell lung cancer (NSCLC) therapeutics, which accounted for 27% of the FDA approvals.
Out of 22 tracked biopharma approvals during the month, seven targeted the indication, including two molecular entities: Tabrecta (capmatinib, Novartis AG) and Retevmo (selpercatinib, Eli Lilly & Co.). The latter was also approved for medullary thyroid cancers.
As a whole, more than half of the approvals – 55% – went to oncology drugs and biologics.
A total of four therapeutic NMEs won FDA approval in May. In addition to Tabrecta and Retevmo were Qinlock (ripretinib, Deciphera Pharmaceuticals Inc.), a kinase inhibitor, for advanced gastrointestinal stromal tumors (GIST), and intravenous artesunate (Amivus LLC), an artemisin derivative, to treat severe malaria.
Four other therapies given the go-ahead for NSCLC are all supplemental approvals and include: Alunbrig (brigatinib, Takeda Pharmaceutical Co. Ltd.), an anaplastic lympohoma kinase tyrosine kinase inhibitor, for first-line ALK-positive metastatic NSCLC; the combination of Opdivo (nivolumab) and Yervoy (ipilimumab), monoclonal antibodies targeting PD-1 and CTLA4 from New York-based Bristol Myers Squibb Co., for treating first-line metastatic or recurrent NSCLC with no EGFR or ALK genomic tumor aberrations; Cyramza (ramuciramab, Eli Lilly and Co.), for use in combination with Tarceva (erlotinib) for NSCLC; and PD-L1 inhibitor Tecentriq (atezolizumab, Genentech Inc.) for treating adults whose metastatic NSCLC tumors have high PD-L1 expression with no EGFR or ALK genomic tumor aberrations.
Tecentriq also was approved for use in combination with Avastin (bevacizumab) for unresectable or metastatic hepatocellular carcinoma.
Other cancer therapies cleared for marketing in the U.S. were two PARP inhibitors for prostate cancer: Rubraca (rucaparib, Clovis Oncology Inc.) and Lynparza (olaparib, Astrazeneca plc/Foundation Medicine Inc.), both for metastatic disease.
Some therapies outside of oncology that were approved in May include Marlborough, Mass.-based Sunovion Pharmaceuticals Inc.’s apomorphine sublingual film (APL-130277), a dopamine agonist for motor fluctuations or off episodes associated with Parkinson’s disease; San Diego-based Evofem Biosciences Inc.’s Amphora, a vaginal pH regulator for the prevention of pregnancy; and Bridgewater, N.J.-based Menlo Therapeutics Inc.’s Zilxi (minocycline, 1.5% topical foam), a tetracycline antibiotic for rosacea.
Two companies received complete response letters during the month. Cambridge, Mass.-based Blueprint Medicines Corp. halted development of its KIT kinase inhibitor, Avyvakit (avapritinib), following a CRL for its use in treating fourth-line GIST, the indication for which Qinlock was approved around the same time. Ayvakit is marketed to treat unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation and will continue in development for systemic mastocytosis. Aside from Blueprint, Silver Spring, Md.-based United Therapeutics Corp. also received a CRL for Trevyent (treprostinil) to treat pulmonary arterial hypertension.
BioWorld tracked a total of 221 regulatory news items in May, up from 201 the prior month. From the American Society of Clinical Oncology (ASCO) meeting held virtually this year due to the COVID-19 pandemic, there were another 106 items of clinical data. Within the regulatory data, there were 47 entries focused on therapeutics or vaccines for COVID-19.
Nevertheless, the virus continues to disrupt business plans. For example, PTC Therapeutics, of South Plainfield, N.J., delayed the filing to the second half of 2020 for a BLA of its gene therapy to treat aromatic L-amino acid decarboxylase deficiency, citing COVID-19 as the reason. BioWorld has tracked 418 clinical trials of biopharma therapeutics sponsored by industry that the pandemic has directly affected.
May regulatory data, as well as clinical results presented through ASCO, can be found at the following links:
Biopharma financings data for 2020 through June 18 can be found here: