FDA accelerates approval for Orland Park newborn's unique genetic illness therapy
In a significant development, the U.S. Food and Drug Administration (FDA) has announced it will approve the Forzinity (elamipretide) injection, a treatment for an ultra-rare disease called Barth syndrome. This decision comes after the FDA set a goal date of September 26, 2025, for the accelerated approval of elamipretide.
The approval process was fast-tracked following an initial report by ABC7 on the importance of elamipretide for cases like that of baby Adel Mohammed, who was rushed to the hospital in respiratory distress when he was not much more than a month old. Adel, who hails from the Chicago area, has been diagnosed with Barth syndrome, an ultra-rare genetic disease that primarily affects males.
Stealth BioTherapeutics, the pharmaceutical company that produces the drug, could have faced potential financial trouble without the FDA's approval. The company's future was precarious, as the drug is currently used to treat Barth syndrome, a condition with approximately 150 diagnosed cases nationwide.
Mohammed's mother, Nour Shaban, has witnessed her son going into cardiac arrest while hospital staff were trying to intubate him. However, with the daily injections of elamipretide for several months, Mohammed has shown improvements, such as sitting up and lifting his head on his own.
Without treatment, Barth syndrome sufferers can exhibit muscle weakness, heart failure, and delayed growth, with most early deaths occurring in infancy. The approval of Forzinity offers hope for families like Mohammed's, who are battling this rare disease and seeking a better life for their loved ones.
A family in Orland Park spoke with ABC7 last month about the drug's importance. The family's name, however, was not provided in the available information. The FDA's official announcement of the accelerated approval of elamipretide was not specified in the text.
Despite the FDA's previous delay in approval due to low sample sizes, the decision to fast-track the process highlights the potential benefits of elamipretide for patients like Adel Mohammed. The approval of Forzinity marks a significant step forward in the treatment of Barth syndrome and could pave the way for further research and developments in the field.
Read also:
- Comprehensive Overview of Addressing Traumatic Brain Injuries (TBIs)
- Enhanced Health Services Provisioned by San Diego Academic Health Partnership Continues During COVID-19 and Beyond
- Vaccination drive targeting infants under 6 months old against bronchiolitis in the region of Andalucia
- Biopsy Basics: Objectives, Varieties, and Potential Hazards - Healthline Illuminated
