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 EMA has given initial endorsement to a new approach to early toxicity testing in which live animals will be replaced by virtual counterparts. The virtual control groups will be derived from data generated in animals that have been used as controls in previous studies. The historical database has been brought together in an EU-funded project to which 20 pharma and crop sciences companies contributed. While limited in scope, this is the first time the EMA has formally endorsed the use of a new approach methodology (NAM) to generate data that could eventually be included in an application for marketing approval.
On top of prison sentences, loss of assets and a $2.6 million restitution order issued several years ago, Li Chen and Yu Zhou had their naturalized U.S. citizenship revoked March 30 as a consequence of stealing exosome-related trade secrets from Nationwide Children's Hospital's Research Institute in Ohio – the hospital that had sponsored them when they first came to the U.S. from China on H-1B Specialty Occupation visas.
Wegovy (semaglutide) has passed the notoriously strict cost-effectiveness scrutiny of the U.K. health technology assessment body and will now be reimbursed for the prevention of further serious cardiovascular events in people who have had a heart attack or stroke, or are diagnosed with peripheral arterial disease.
Once again, Amgen Inc.’s Tavneos (avacopan) is under U.S. FDA scrutiny, as the agency alerted patients and doctors March 31 about 76 cases of drug-induced liver injury, including eight deaths, with “reasonable evidence of a causal association” with the drug.
Eli Lilly and Co. anticipates shipping newly approved Foundayo (orforglipron) within the next week, as the drug becomes the second oral weight-loss glucagon-like peptide-1 (GLP-1) receptor agonist to enter the U.S. market following December’s approval of Novo Nordisk A/S’ Wegovy (semaglutide) pill.
Seeking to increase their understanding of the opportunities and challenges of using digital health technologies (DHTs) in drug trials, the U.S. FDA’s CBER and CDER are requesting public feedback to help inform new guidance as the technologies continue to advance.
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.
What do a patent dispute over a CRISPR/Cas system, a rejected whistleblower case involving lab tests and a vaccine injury claim parading as multidistrict tort litigation have in common? All three were denied cert in the U.S. Supreme Court’s latest orders list.
Introduced last year as a pilot program, the U.S. FDA Commissioner’s National Priority Voucher (CNPV) could be here to stay – at least for the duration of Marty Makary’s tenure as FDA commissioner. Since the FDA unveiled the CNPV last June, it has welcomed 18 products from 16 companies into the “game-changer” program for patients, as Makary described it. The goal is to provide an “ultrafast review pathway,” one to two months instead of the standard 10 to 12 months, for drugs and biologics of strategic national importance while maintaining the FDA’s scientific and regulatory standards, according to the agency.
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