Welcome to this week’s edition of the Life Sciences Voice Top Five Newsletter, your go-to source for the latest breakthroughs in the life sciences industry! This time, we’re taking a look at Eli Lilly’s move to counter compounded versions of its popular drug Zepbound in the market, the FDA’s approval of Romvimza for tenosynovial giant cell tumor, MeiraGTx’s gene therapy’s positive impact on vision restoration in children, and other top news! Stay informed and inspired by the innovations driving life sciences forward!
Eli Lilly Reduces Zepbound Prices and Introduces New Dosage Options
Eli Lilly has reduced the price of vials for its obesity drug, Zepbound, and expanded available dosage options. The 2.5-mg and 5-mg vials now cost $349 and $499 per month, respectively, while new 7.5-mg and 10-mg vials are priced at $599 and $699. A special pricing option allows patients to pay $499 for higher-dose refills if they adhere to a 45-day schedule. The move seeks to compete with compounded alternatives and rival medications like Novo Nordisk’s Wegovy. Regulatory changes may impact the availability of compounded versions, as the FDA recently removed Zepbound and Wegovy from its shortage list.
FDA Approves Romvimza for Rare Joint Tumor, Offering New Treatment Option
The FDA has approved Romvimza (vimseltinib) for treating tenosynovial giant cell tumor (TGCT) in patients for whom surgery is not viable. Developed by Ono Pharmaceuticals, Romvimza is a kinase inhibitor that targets the CSF1 receptor to limit tumor growth. Approval was based on Phase III MOTION study results, showing a 40% response rate at 25 weeks. Unlike competitor Turalio, Romvimza requires twice-weekly dosing and lacks a hepatotoxicity warning. Ono acquired the drug through its $2.4 billion purchase of Deciphera Pharmaceuticals and plans to launch it in the U.S. soon. TGCT can cause severe joint impairment if untreated.
MeiraGTx’s investigational gene therapy Restores Vision in Children with Severe Retinal Dystrophy
MeiraGTx’s investigational gene therapy for AIPL1-associated severe retinal dystrophy has led to significant vision improvements in 11 children born legally blind. A Lancet study on four treated children reported measurable gains in visual acuity within four weeks, while untreated eyes continued deteriorating to unmeasurable levels. Additional benefits included improved communication, education, and social interaction. One child developed cystoid macular edema, which partially resolved. The therapy, delivered via a single injection using an adeno-associated virus, is now under expedited regulatory review in the UK, with U.S. FDA discussions ongoing. Seven more children later received treatment in both eyes.
Roche Unveils Sequencing-by-Expansion for Faster, More Precise DNA Analysis
Roche has introduced sequencing-by-expansion (SBX), a patented genetic analysis technique that disassembles DNA and amplifies each base’s signal independently. SBX combines DNA synthesis with nanopore-based molecular reading, aiming to overcome the time constraints and signal discrimination challenges of traditional sequencing. The technology builds on Roche’s acquisitions of Stratos Genomics (2020) and Genia Technologies (2014). SBX stretches DNA up to 50 times its original length, enhancing readability before passing through nanopores for sequencing. While still in development, Roche plans to launch the technology next year, with potential applications in whole-genome, exome, and RNA sequencing.
FDA Approves Mirum’s Ctexli as First Treatment for Rare Genetic Disorder CTX
Mirum Pharmaceuticals secured FDA approval for Ctexli (chenodiol) as the first U.S.-approved treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder that disrupts fat metabolism. The approval follows Mirum’s $210 million acquisition of Travere Therapeutics’ bile acid portfolio in 2023. Clinical studies showed that 250 mg of Ctexli, taken thrice daily, effectively reduced toxic cholesterol metabolites. Due to potential liver toxicity, patients must undergo regular liver function tests. Though chenodiol was available off-label for over a decade, its full approval grants Mirum market exclusivity. Analysts estimate potential revenue between $150 million and $200 million annually.
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