Merck’s subsidiary, SpringWorks Therapeutics, has received marketing authorization from the European Commission (EC) for Ogsiveo (nirogacestat) to address desmoid tumors, which are rare, locally aggressive tumors developing in the body’s connective tissues. Annually, the European Union witnesses between 1,300 to 2,300 new cases of desmoid tumors. Ogsiveo, an oral gamma secretase inhibitor, has been sanctioned as a standalone treatment for adults with advancing desmoid tumors necessitating systemic therapy.
The endorsement of Ogsiveo is founded on results from the Phase III DeFi trial involving 142 adult participants with progressive desmoid tumors. This global, double-blind, randomized, and placebo-controlled study evaluated the safety, effectiveness, and tolerability of nirogacestat in adults. The primary endpoint was met by revealing an enhancement in progression-free survival (PFS). Furthermore, Ogsiveo exhibited a considerable 71% decrease in the risk of disease progression in contrast to the placebo group.
Additionally, the Ogsiveo group displayed a significantly higher objective response rate (ORR), with 41% of individuals achieving a confirmed response, compared to only 8% in the placebo cohort. Merck Healthcare’s CEO and executive board member, Danny Bar-Zohar, emphasized the importance of expanding Ogsiveo’s benefits to European patients, following its already established status as the standard systemic therapy for desmoid tumors in the US. This approval aligns with Merck’s commitment to the rare tumor patient community, especially after the recent EC approval for their therapy targeting NF1-PN patients.
In the US, Ogsiveo has already been sanctioned for treating adults with progressing desmoid tumors. Both the US Food and Drug Administration and the European Medicines Agency have recognized Ogsiveo as an orphan drug for this specific indication, highlighting its significance in addressing unmet medical needs. The successful EC approval of Ogsiveo underscores Merck’s dedication to advancing innovative treatments for rare diseases and solidifies its position as a leader in the biopharmaceutical industry.
The announcement of the EC approval for Ogsiveo was initially reported by Pharmaceutical Technology, a brand under GlobalData. It is essential to note that the information presented serves as general informational purposes and should not be solely relied upon for decision-making. Professional or specialist advice should always be sought before initiating or abstaining from any actions based on the content provided.
Key Takeaways:
– Ogsiveo (nirogacestat) by Merck KGaA’s subsidiary, SpringWorks Therapeutics, has received EC approval for treating desmoid tumors in the European Union.
– The approval is based on positive results from the Phase III DeFi trial, demonstrating improved progression-free survival and a significant decrease in disease progression risk.
– Ogsiveo has already been established as the standard systemic therapy for desmoid tumors in the US and is designated as an orphan drug by regulatory authorities.
– Merck’s commitment to rare disease patients is reflected in its efforts to provide innovative treatments like Ogsiveo to address unmet medical needs effectively.
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