Trading of Sarepta Therapeutics Inc. shares (NASDAQ:SRPT) was halted while the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (OTAT) sorted data regarding the gene transfer therapy delandistrogene moxeparvovec in ambulatory patients with Duchenne muscular dystrophy. OTAT panelists will vote this afternoon on whether to recommend a green light for SRP-9001. Research with the “one-and-done” therapy used, as a surrogate endpoint, expression of Sarepta’s microdystrophin at week 12 after administration.

Despite IPO drought, biopharma financings tracking ahead of last year

Biopharma financings are pacing more than $300 million higher than at the end of April last year, but are well behind the total through April 2021 when the value of financings more than doubled any recent year due to the response to the COVID-19 pandemic. Through the end of April, biopharmas have raised $19.83 billion this year, compared to $17.5 billion through the same month in 2022, $53.7 billion in 2021, $24.04 billion in 2020 and $19.53 billion in 2019. In terms of volume, the 365 financing deals this year are more than 2022's 338 through the end of April. This year is nearly on par with 2019's 367 financings through the month, but tracking far less than 2020's 448 and 2021's 645 deals. 

Armed with new tools, US FDA picks up pace of accelerated approvals 

Despite congressional concerns about accelerated approval, the U.S. FDA’s use of the pathway is not slowing down. If anything, it’s picked up pace since Congress gave the agency stronger authority last year to monitor drugs approved based on a surrogate endpoint and to ensure that confirmatory trials are progressing in a timely way. Of the 13 novel drugs the FDA approved from January through March, four, or nearly 31%, were granted accelerated approval. In comparison, the agency greenlighted only one novel drug for accelerated approval in the first quarter of 2022. While much of that has to do with the flow and timing of the drug pipeline, it does show the FDA isn’t reserving the accelerated pathway for a few isolated drugs.

BIO Korea 2023: Korean ecosystem offers advantages for clinical differentiation in hot areas like ADC

Korean biopharmas in the antibody-drug conjugate (ADC) space can leverage the strength of the Korean ecosystem when partnering with global pharma companies, said investors during the BIO Korea 2023 conference in Seoul on May 12. Just as in the global space, Korean firms are moving in on the growing ADC space, and although many of them have good antibodies, chemistry or linker technology, no company has all three technologies; hence, the need to partner. 

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Abeona, Ani, Bellerophon, Biomx, Cassava, Cytoagents, Cytokinetics, Extend, First Wave, Immunitybio, Innovent, Lundbeck, Mindbio, Mirum, Mithra, Navidea, Neurobo, Olema, Onconova, Oniria, Otsuka, Palisade, Pan Cancer T, Praxis, Salarius, Soligenix, Sunshine, Theravance, Vir