“We’re going to battle,” PTC Therapeutics Inc. CEO Matthew Klein said, responding to a surprise negative opinion from the EMA’s Committee for Medicinal Products for Human Use on converting the conditional marketing authorization to full status for Translarna (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy. The opinion applies to the renewal of the existing conditional authorization, too.
Supporting their conclusions with data from the same phase III study, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion for extending the use of Oncopeptides AB’s Pepaxti (melflufen) to earlier lines of treating relapsed, refractory multiple myeloma even as the FDA dug in its heels about withdrawing the drug from the U.S. market.
As part of its quest to advance its three first-in-class heart failure drug candidates toward the clinic, Corteria Pharmaceuticals SAS has closed an oversubscribed €65 million (US$70.7 million) series A, co-led by investors Orbimed and Jeito Capital, companies based in the U.S. and Europe, respectively.
Blood biomarkers have been found in patients hospitalized with acute COVID-19 that are predictive of the cognitive defects of long COVID. Post COVID-19 deficits in cognition, including brain fog, are common and debilitating. They are also clinically complex, with both objective and subjective components. In the U.K., one in eight patients received their first ever neurological or psychiatric diagnosis within six months following COVID-19.
Cochlear Ltd. and Sensorion SA have published encouraging results on the combination of a molecule with a cochlear implant. Their proof-of-concept phase 2a study involved the combination of an orphan drug recognized in both Europe and the U.S. with cutting edge hearing aid implants to enhance the auditory capabilities of hearing aid users.
A recent bipartisan request for funding of a study on replacing U.S. drug patents with cash prizes is just one more symptom of a larger global malady that makes patents the scapegoat for bigger problems that have nothing to do with intellectual property (IP), David Kappos, board co-chair of the Council for Innovation Promotion (C4IP), told BioWorld.
Building on capabilities developed during the COVID-19 pandemic, the U.K. Health Security Agency’s Vaccine Development and Evaluation Centre hit the ground running.
Calling it a “landmark judgment,” the U.K.’s Competition and Markets Authority welcomed the Competition Appeal Tribunal’s Aug. 8 unanimous decision upholding an £84 million (US$107 million) fine levied against London-based Advanz Pharma Corp. for excessive drug pricing.
A logjam of clinical trial applications that has bult up at the U.K. Medicines and Healthcare products Agency (MHRA) is deterring companies from conducting studies in the U.K., according to the Bioindustry Association.
Despite a newly signed $645 million commercialization deal with Neuraxpharm Group for multiple sclerosis drug Briumvi (ublituximab) in Europe, shares of TG Therapeutics Inc. (NASDAQ:TGTX) tanked by 49.3% on Aug. 1 due to a perceived miss in U.S. net sales for the anti-CD20 monoclonal antibody during its first full quarter on the U.S. market.
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