The U.S. Food and Drug Administration (FDA) has unveiled two major medical advancements, including the approval of a groundbreaking gene therapy for a rare eye condition and the expansion of a nasal spray treatment for life-threatening allergic reactions in young children. These updates reflect the FDA’s ongoing dedication to enhancing patient care and providing innovative solutions for various health challenges.
FDA Approves Encelto for MacTel Treatment
On March 5, the FDA granted approval to Encelto (revakinagene taroretcel-lwey), an innovative allogeneic encapsulated cell-based gene therapy, to treat adults with idiopathic macular telangiectasia type 2 (MacTel). This rare and progressive disease affects the macula, a critical part of the retina that is vital for sharp central vision, and causes the degeneration of photoreceptor cells responsible for light detection.
Encelto offers a unique treatment option, utilizing a one-time surgical intervention performed by an ophthalmologist. The therapy works by delivering recombinant human ciliary neurotrophic factor, which may help preserve the survival and function of macular photoreceptors. This breakthrough provides a promising solution for MacTel patients, who previously had limited options to manage this debilitating eye disease.
Neffy Nasal Spray Approved for Younger Children
In a separate announcement, the FDA expanded the approval for neffy (epinephrine nasal spray) on March 5, now including children aged four years and older who weigh between 15 and 30 kilograms (approximately 33 to 66 pounds). Initially approved in 2024, neffy is the first FDA-authorized nasal spray designed to treat anaphylaxis—an acute, severe allergic reaction—without the need for an injection.
The updated approval now extends this needle-free alternative to a younger and lighter pediatric population. Each dose of neffy contains 1 mg of epinephrine, providing a safe and effective emergency treatment for allergic reactions triggered by food, insect stings, or medication. While common side effects include nasal discomfort, congestion, and dry throat, these effects mirror those experienced by older children and adults, reinforcing the safety profile of the nasal spray.
A Commitment to Patient-Centered Care
The FDA’s latest approvals underscore its commitment to advancing patient-centered care by supporting therapies that address critical health needs. Encelto offers a new hope for individuals suffering from the vision loss caused by MacTel, while the expanded use of neffy ensures that more children can access a life-saving, non-invasive treatment for anaphylaxis. Both decisions demonstrate the FDA’s continued focus on medical innovation and its role in improving the lives of patients and their families.
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