According to the PDUFA schedule, the U.S. Food and Drug Administration (FDA) is likely to make a review decision on four new drugs in the last two weeks of April, two of which are from China.
Liu Yingqi cartographed
1、Mavacamten
Company Name: Bristol-Myers Squibb
Indications: Obstructive hypertrophic cardiomyopathy
Mavacamten is an oral myocardial myoglobulin inhibitor acquired by Bristol-Myers Squibb in November 2020 with the acquisition of MyoKardia for oral treatment of obstructive hypertrophic cardiomyopathy (oHCM). The drug's NDA, submitted in March 2021, was received by the FDA based on positive Phase 3 clinical results, which showed that both primary and secondary efficacy endpoints were met and that the safety was similar to that of placebo. If approved, it could become a potential "first-in-class" drug for the disease mechanism of oHCM. In addition to listing applications in the United States, Mavacamten also has a listing authorization application under review in the European Union for the same indications. Currently, the company is conducting clinical trials to evaluate mavacamten's potential ability to treat other indications, including non-obstructive hypertrophic cardiomyopathy.
2、 AXS-07
Company name: Axsome Therapeutics
Indications: Migraine
Focused on the treatment of central nervous system disorders, Axsome is developing AXS-07, a novel, rapidly absorbing combination of oral fixed doses with the active ingredients meloxicam and rizatriptan, and using Axsome's MoSEIC formulation technology for the acute treatment of migraines. Positive results from two Phase 3 trials showed rapid and sustained efficacy compared with placebo and active control rizatriptan.
3. Sofotinib
Company Name: Hehuang Pharmaceutical
Indications: Pancreatic and non-pancreatic neuroendocrine tumors
Hehuang Medicine's suvaftinib is an oral tyrosine kinase inhibitor with dual activity of anti-angiogenesis and immunomodulatory for the treatment of pancreatic and non-pancreatic neuroendocrine tumors (NET). Based on positive Phase 3 results, Surufatinib's NDA application was accepted by the FDA in June 2021, and the results showed that the drug had reached the primary efficacy endpoint. The drug is already available in China and is also seeking approval from the European Union. If approved in the U.S. under current plans, the company expects the drug to be available in the second half of 2022, providing additional treatment options for patients with advanced NET.
4. Toripalimab
Company Name: Coherus BioSciences
Indications: Nasopharyngeal carcinoma
Toripalimab, jointly developed by Junshi Bio and CoherusBioSciences, is an anti-PD-1 monoclonal antibody used to treat recurrent or metastatic nasopharyngeal carcinoma. In November 2021, the FDA received a Biological Product Licensing Application (BLA) from Toripalimab, supported by positive safety and efficacy results from Phase 2 and Phase 3 studies, and was granted priority review qualification. Triprilizumab has been approved for marketing in China for the treatment of non-resectable or metastatic melanoma. For advanced nasopharyngeal cancer, there is currently no FDA-approved immuno-oncology therapy. (Liu Yingqi)
