As part of its required series of guidances on using real-world evidence, the U.S. FDA released a draft guidance in response to sponsors’ growing interest in the potential use of observational studies to contribute to a demonstration of the effectiveness or safety of a drug or biologic.
Although there’s bipartisan interest in the U.S. Congress to hold pharmacy benefit managers (PBMs) accountable for their contribution to the costliest drug prices in the world, the Biden administration ignored PBMs when it again focused on drug companies as the bad guys of pricing in its proposed 2025 budget.
After months of wrangling, the update of the EU pharmaceutical legislation passed an important milestone on March 19, when members of parliament on the health committee reconciled their opposing views and voted the file through.
Two days after the U.S. FDA announced approval of gene therapy Lenmeldy (atidarsagene autotemcel), making it the first treatment option for rare disease metachromatic leukodystrophy, Orchard Therapeutics and parent firm Kyowa Kirin Co. Ltd. disclosed the wholesale acquisition price of $4.25 million for the one-time treatment, which edges out hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to become the world’s most expensive drug.
Scottsdale, Ariz.-based Renovacare Inc., has come to terms over allegations that the company engaged in what the Securities and Exchange Commission (SEC) said was a pump-and-dump scheme designed to defraud the company’s investors. The initial SEC complaint dated May 28, 2021, alleges that Renovacare and company executives began making misleading statements to investors as far back as July 2017, making this yet another cautionary tale of duplicitousness in life science startups.
Vicore Pharma Holding AB was granted a U.S. FDA breakthrough device designation for Almee, a cognitive behavioral therapy (CBT) software, to be used as an adjunct treatment by patients with anxiety symptoms due to pulmonary fibrosis. Vicore developed the nine-week CBT digital therapy in partnership with Alex Therapeutics AB.
The U.S. FDA has followed up on reports of problematic syringes made by several companies in mainland China, recommending that U.S. suppliers, consumers and health care organizations stop using these products unless no alternatives are available. The agency said it has issued warning letters to three of these companies, at least one of which appears to have been the supplier of Monoject syringes that have been the subjects of recent FDA recalls.
About six months after Johnson & Johnson (J&J) returned rights to the compound, oral Tryvio (aprocitentan) won FDA clearance for Idorsia Ltd. Given once daily at a 12.5 mg dose, Tryvio is indicated for hypertension in combination with other blood pressure drugs in patients whose condition is not adequately controlled.
An MHRA investigation that began in the U.K. 16 years ago has concluded with Kamlesh Vaghjiani, a former director of Kappin Ltd., being sentenced to concurrent prison sentences of eight and seven months, both of which are suspended for a year and a half.
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