WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has announced significant advancements in medical treatments, including the approval of a novel gene therapy for a rare eye disease and an expanded indication for a nasal spray designed to treat life-threatening allergic reactions in young children. These updates reflect the FDA’s continued commitment to providing innovative and accessible treatments for a wide range of medical conditions.
Approval of Encelto for Idiopathic Macular Telangiectasia Type 2 (MacTel)
On March 5, the FDA approved Encelto (revakinagene taroretcel-lwey), a groundbreaking allogeneic encapsulated cell-based gene therapy for the treatment of adults with idiopathic macular telangiectasia type 2 (MacTel). This rare, progressive disease affects the macula—a part of the retina essential for sharp central vision—and leads to the degeneration of photoreceptor cells responsible for detecting light.
Encelto offers a one-time surgical intervention performed by a qualified ophthalmologist. The therapy works by delivering recombinant human ciliary neurotrophic factor, which may support the survival and function of macular photoreceptors. This innovative approach marks a major step forward for patients with MacTel, who previously had limited treatment options for managing this debilitating condition.
Expanded Indication for Neffy Nasal Spray
Also on March 5, the FDA announced an expanded indication for neffy (epinephrine nasal spray) to include children aged four years and older who weigh between 15 and less than 30 kilograms (approximately 33 to 66 pounds). Approved in 2024, neffy is the first FDA-authorized nasal spray designed to treat anaphylaxis—a severe and potentially fatal allergic reaction—without the need for an injection.
Previously approved for use in adults and children weighing at least 30 kilograms (66 pounds), the expansion now makes the treatment available to a younger and lighter pediatric population. Each dose consists of a 1 mg spray of epinephrine, offering a needle-free alternative for managing emergencies such as severe allergic responses to food, insect stings, or medication.
Common side effects for this new demographic include nasal discomfort, congestion, dry throat, nasal dryness, and tingling. These effects mirror those observed in older populations, helping reinforce the safety profile of this novel delivery method.
Patient-Centered Innovation
The FDA’s recent decisions aim to advance patient care by supporting innovative treatments that address unmet medical needs. Encelto represents a promising option for patients suffering from the progressive loss of vision caused by MacTel, while the expanded indication for neffy ensures more children have access to life-saving, needle-free anaphylaxis treatment. Both announcements illustrate how medical innovation continues to address critical health challenges, offering hope and solutions for patients and their families.
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