Orally delivered Tryvio (aprocitentan) gained U.S. FDA clearance for Idorsia Ltd. to treat hypertension in combination with other blood pressure drugs in patients whose condition is not adequately controlled on available therapies. Rights to the compound were handed back to Idorsia last September by Johnson & Johnson. CEO Jean-Paul Clozel said the FDA nod leaves Allschwil, Switzerland-based Idorsia in “a very clear situation” with regard to finding another partner. Pricing of Tryvio was not disclosed.

Move over, Hemegenix: Lenmeldy tops price list at $4.25M

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 (MLD), 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. Orchard, which has previously launched the therapy in Europe and other areas, is activating five treatment centers for treating MLD, a disease believed to affect fewer than 40 children annually in the U.S.

EU’s largest pharma reform in 20+ years advances, despite concerns

After months of wrangling, the update of the EU pharmaceutical legislation passed an important milestone on March 19, when members of parliament (MEPs) on the health committee reconciled their opposing views and voted the file through. The agreed position addresses some, but not all of the industry’s objections to the original proposal put forward by the European Commission in April 2023. A particular source of grievance was the proposal to reduce the length of regulatory data protection, during which competitors cannot access clinical trial data submitted with marketing applications, from eight years currently, to six. In the event, MEPs voted for a minimum regulatory data protection period of 7.5 years, plus a further two years when generic or biosimilar products cannot be put on the market.

US senators: Want lower drug prices? Reform PBMs

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. Sen. Mike Crapo (R-Idaho), ranking member of the Senate Finance Committee, noted the administration’s singular focus in his opening remarks at a hearing last week on the proposed Health and Human Services (HHS) budget. The budget “makes virtually no mention of the robust bipartisan, bicameral efforts to reform PBM practices, instead opting to double down on price control policies that polarize members in both chambers,” Crapo told HHS Secretary Xavier Becerra. Meanwhile, nearly two dozen Democratic and Republican senators signed on to a letter calling on Senate leadership to take immediate action on enacting PBM reforms to reduce the cost of prescription drugs.

With $150M in hand, Clasp launches with T-cell engagers

Clasp Therapeutics Corp., a new company that uses modular T-cell engagers to treat cancer, has launched with a $150 million financing. The therapy is tailored to a patient’s individual immune system that is directed to oncogenic driver mutations. The off-the-shelf, antibody-like therapies are designed to treat a variety of hard-to-treat tumor types. The science comes from research performed at Johns Hopkins University. The financing was led by Catalio Capital Management, Third Rock Ventures and Novo Holdings, with participation from Vivo Capital, Cure Ventures, Blackbird Bioventures, Pictet Alternative Advisors, American Cancer Society’s Bright Edge and Alexandria Venture Investments.

Also in the news

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