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 looking at the FDA’s approval for Merck’s RSV treatment in infants, Sanofi’s acquisition of Blueprint Medical Corporation, why the FDA has halted Rocket Pharmaceuticals’ gene therapy trial, and more! Stay informed and inspired by the innovations driving life sciences forward.
FDA Approves Merck’s Enflonsia for RSV Prevention in Infants
The FDA has approved Merck’s monoclonal antibody clesrovimab, marketed as Enflonsia, for preventing RSV-related lower respiratory tract disease in infants during their first RSV season. Enflonsia, priced at $556 per dose, is administered once to full-term, pre-term, and at-risk infants. A late-stage trial showed an 84% reduction in RSV-related hospitalizations and a 60% decrease in medically attended infections. Enflonsia competes with Sanofi and AstraZeneca’s Beyfortus and differs by targeting a distinct antigenic site. Merck plans delivery ahead of the 2025–2026 season. The CDC’s upcoming review may be affected by the recent dismissal of all its panel members.
Sanofi to Acquire Blueprint Medicines in $9.5 Billion Deal, Focus on Rare Immunological Diseases
Sanofi has announced its plan to acquire U.S.-based Blueprint Medicines for up to $9.5 billion, the largest healthcare acquisition in Europe this year, according to LSEG data. The deal centers on Blueprint’s systemic mastocytosis treatment, Ayvakit, and a pipeline of immunology candidates. Sanofi will pay $129.00 per share and offer contingent value rights tied to BLU-808 milestones. The acquisition includes elenestinib and expands Sanofi’s presence in the immunology field. It follows recent deals, including the acquisitions of Vigil Neuroscience and Inhibrx. Sanofi expects to finalize the Blueprint transaction in Q3 without altering its 2025 financial guidance.
FDA Halts Rocket’s Danon Disease Gene Therapy Trial After Patient Death
The FDA has issued a clinical hold on Rocket Pharmaceuticals’ Phase 2 gene therapy trial for Danon disease after a patient died from complications following treatment with RP-A501. The patient developed capillary leak syndrome one week post-infusion and later succumbed to an infection. Two patients had received a revised protocol including a C3 inhibitor; neither showed thrombotic microangiopathy, but both developed capillary leak syndrome. Rocket halted dosing and notified the FDA. The company is now revising its data timeline to mid-2026 and has extended its cash runway. The hold does not affect other Rocket pipeline programs.
RegenXBio Reports Functional Gains in RGX-202 DMD Gene Therapy Trial
RegenXBio released updated results from its Phase 1/2 trial of RGX-202 for Duchenne muscular dystrophy (DMD), showing functional improvements in five boys aged 6–12 treated at the pivotal dose. At nine and 12 months post-treatment, patients demonstrated gains in time to stand, 10-meter walk-run, and time to climb tests, exceeding pre-treatment baselines and expected disease progression. Four of five patients reached the 12-month mark. No serious adverse events were reported. Compared to an earlier lower-dose group, gains were slightly lower, possibly due to age differences. The company aims to complete enrollment this year and seek approval in 2026.
Insmed Reports Positive Phase 2b Results for TPIP in Pulmonary Arterial Hypertension
Insmed announced results from a Phase 2b trial of treprostinil palmitil inhalation powder (TPIP) for pulmonary arterial hypertension (PAH), showing a 35% placebo-adjusted reduction in pulmonary vascular resistance and a 35.5-meter improvement in six-minute walk distance among 102 participants. The once-daily inhaled therapy also reduced a heart stress-related protein by 60%. Cough was the most frequent adverse event, affecting 40.6% of patients. Insmed plans to begin Phase 3 trials in early 2026 and conduct a separate study in PH-ILD before the end of 2025. The company’s stock rose over 27% following the announcement of these findings.
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