Replimune Group Inc. received another complete response letter (CRL) from the U.S. FDA for immunotherapy vusolimogene oderparepvec to treat advanced melanoma, sending shares plunging by more than 19% on the April 10 PDUFA date.
Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).
The U.S. path forward is narrowing for Vanda Pharmaceuticals Inc.’s tradipitant as a treatment for gastroparesis, a serious disorder for which there’s been no new treatment in several decades.
It’s déjà vu all over again for Aldeyra Therapeutics Inc., which disclosed its third complete response letter (CRL) for dry eye disease candidate reproxalap, with the U.S. FDA citing a lack of substantial and consistent evidence of efficacy.
Vanda Pharmaceuticals Inc. will get to take its argument for twice-rejected jet lag disorder drug Hetlioz (tasimelteon) before the U.S. FDA in a formal evidentiary public hearing, a rare move by the agency that the firm claims underscores the “gravity of the legal and scientific issues” it has raised.
Though it’s largely viewed by analysts as a simple delay rather than a setback, Disc Medicine Inc.’s unexpected complete response letter (CRL) for bitopertin in the rare genetic disorder erythropoietic protoporphyria (EPP) raises more questions regarding consistency and stability at the U.S. FDA.
As many had predicted following news of a clinical hold on the gene therapy last month, Regenxbio Inc. disclosed receipt of a complete response letter (CRL) regarding its BLA for RGX-121 (clemidsogene lanparvovec) in mucopolysaccharidosis II, an ultrarare neurodegenerative disease in dire need of new therapies.
It’s not often that disclosure of a complete response letter (CRL) causes a company’s share price to rise, but that was case for Aquestive Therapeutics Inc., which saw its stock (NASDAQ:AQST) rise 39% to close Feb. 2 at $4.10 on news the U.S. FDA has declined – for now – to approve the NDA for Anaphylm (dibutepinephrine), delaying the commercial entry of what could be the first oral alternative to Epipen.
Nader Pourhassan, the former president and CEO of Cytodyn Inc., was sentenced Jan. 23 to 30 months in prison for his role in a securities fraud scheme to deceive investors about the Vancouver, Wash.-based company’s development of leronlimab as a treatment for HIV and COVID-19.
A second complete response letter issued by the U.S. FDA for Atara Biotherapeutics Inc.’s allogeneic T-cell immunotherapy Ebvallo (tabelecleucel) for Epstein-Barr virus positive post-transplant lymphoproliferative disease is “a complete reversal of position” by the agency, which had previously confirmed the single arm Allele trial was enough to support a BLA under the accelerated approval pathway, the company said.
