Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), today announced The Medicines and Healthcare products Regulatory Agency (MHRA) authorization of NULIBRY.
NULIBRY is the first and only treatment in Great Britain for patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about four years The Medicines and Healthcare products Regulatory Agency’s (MHRA) decision is based on efficacy and safety data collected to date SOLANA BEACH, Calif., April 16, 2024 /PRNewswire/ -- Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), today announced The Medicines and Healthcare products Regulatory Agency (MHRA) authorization of NULIBRY® (fosdenopterin) for Injection as the first therapy for the treatment of patients in Great Britain (GB) with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants. MoCD Type A is known to impact fewer than 150 patients globally with a median survival age of four years. NULIBRY is a first-in-class synthetic cPMP substrate replacement therapy that was approved by the U.S. Food and Drug Administration (FDA) in 2021 to reduce the risk of mortality in patients with MoCD Type A. Following this decision by the MHRA, NULIBRY is the first and only approved therapy in GB for MoCD Type A. “The MHRA approval of NULIBRY opens the door for healthcare providers in Great Britain to utilize this innovative treatment to meet the previously unmet needs of patients and their caregivers,” said Matt Heck, President & Chief Executive Officer of Sentynl. “This approval advances our mission to make a positive impact in the lives of patients suffering from rare diseases, especially one as devastating as MoCD Type A.” NULIBRY’s MHRA approval was supported by data from three clinical trials that demonstrated the safety and efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that NULIBRY-treated patients had a 5.5 times lower risk of death than that of the untreated patients. Moreover, the survival probability at 3 years of age was 85.5% for NULIBRY-treated patients and 55.1% for untreated control patients. In March 2022, Sentynl acquired the global rights to NULIBRY from BridgeBio Pharma, Inc. and is responsible for the ongoing development, manufacturing and commercialization of fosdenopterin globally. For questions on continued access to NULIBRY, please contact the Sentynl Cares support team at 1-888-251-2800. About Molybdenum Cofactor Deficiency (MoCD) Type A MoCD Type A is an ultra-rare disease. The incidence and prevalence of MoCD Type A in Great Britain are not known, but the estimated prevalence is 0.005 per 10,000. Based on these estimates, MoCD Type A is likely to be underdiagnosed. The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment. About NULIBRY® (fosdenopterin) for Injection References Important Safety Information Warnings and Precautions Potential for Photosensitivity NULIBRY can make the patient oversensitive to sunlight. NULIBRY-treated patients or their caregivers are advised to avoid or minimize patient exposure to sunlight and artificial UV light and adopt precautionary measures when exposed to the sun, including wearing protective clothing and sunglasses, and use broad-spectrum sunscreen with high SPF in patients 6 months of age and older. If photosensitivity occurs, caregivers/patients are advised to seek medical attention immediately and consider a dermatological evaluation. Adverse Reactions The most common adverse reactions in NULIBRY-treated patients were infusion catheter–related complications (89%), pyrexia (fever) (78%), viral infection (56%), pneumonia (44%), otitis media (ear infection) (44%), vomiting (44%), cough/sneezing (44%), viral upper respiratory infection (33%), gastroenteritis (33%), diarrhea (33%) , and bacteremia (33%). Adverse reactions for rcPMP-treated patients were similar to the NULIBRY-treated patients. Patient Counseling Information Please read the FDA-approved NULIBRY Prescribing Information and Instructions for Use and follow the instructions on how to prepare and administer NULIBRY. NULIBRY has a potential for photosensitivity; see Warnings and Precautions. Seek medical attention immediately if the patient develops a rash or if they notice symptoms of photosensitivity reactions (redness, burning sensation of the skin, blisters). You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. About Sentynl Therapeutics About Zydus Group View original content to download multimedia:https://www.prnewswire.com/news-releases/sentynl-therapeutics-receives-mhra-authorization-of-nulibry-fosdenopterin-for-treatment-of-mocd-type-a-in-great-britain-302117194.html SOURCE Sentynl Therapeutics |