BridgeBio Pharma, Sentynl Therapeutics and Medison Pharma Announce Approval in Israel for NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A
Thursday, August 11, 2022
BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), along with partner Medison Pharma, a global pharmaceutical company focused on providing access to highly innovative therapies to patients in international markets, today announced that the State of Israel Ministry of Health has approved NULIBRY® (fosdenopterin) for Injection as the first therapy in Israel to treat MoCD Type A with the indication to reduce the risk of mortality for patients with molybdenum cofactor deficiency (MoCD) Type A. MoCD Type A is an ultra-rare and progressive condition, known to impact less than 150 patients globally with a median survival of four years.
"At BridgeBio, we believe that no disease is too rare to address. We are grateful that NULIBRY is now an approved therapy in Israel, and we are focused on ensuring that even more children around the world who suffer with MoCD Type A have access to this treatment," said BridgeBio founder and CEO Neil Kumar, Ph.D.
NULIBRY is a cPMP substrate replacement therapy that was approved by the U.S. FDA in February 2021 to reduce the risk of mortality in patients with MoCD Type A. In July 2022 the CHMP recommended that NULIBRY be approved in the EU as a treatment for patients with MoCD Type A. Based on the CHMP recommendation, a decision by the European Commission (EC), which authorizes marketing applications in the EU, is expected on the NULIBRY application later this year.
"The approval of NULIBRY in Israel is a promising development for children with MoCD Type A. At Zydus and Sentynl we are driven by the purpose of empowering people with the freedom to live healthier and more fulfilled lives. NULIBRY will bring us closer to realizing our purpose and making a meaningful difference in the lives of children with this rare disorder," said Dr. Sharvil Patel, Managing Director of Zydus Lifesciences, the parent company of Sentynl Therapeutics.
"We are very pleased to share the news of the Israeli approval on the heels of receiving the CHMP's recommendation in favor of NULIBRY. We are confident this is only the beginning for patients living with MoCD Type A and we intend to continue advancing our efforts to broaden access to this therapy throughout the world for children in need, while increasing awareness of a disease that has historically been underdiagnosed," said Matt Heck, CEO of Sentynl.
The approval by the State of Israel Ministry of Health is supported by data from three clinical trials that demonstrated 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 reduced the risk of death by 82% and increased the probability of survival to 84% at three years compared to 55% in the untreated, genotype-matched, historical control group in the natural history study.
Animal studies have identified that NULIBRY has phototoxic potential. In the clinical trials, the most common adverse reactions reported in two or more NULIBRY-treated patients with MoCD Type A were catheter-related complications (89%), pyrexia (fever; 78%), viral infection (56%), pneumonia (44%), otitis media (ear infection; 44%), vomiting (44%) and cough/sneezing (44%). Adverse reactions for the rcPMP-treated patients were similar to the NULIBRY-treated patients.
"We are proud that our partnership with BridgeBio Pharma and Sentynl enables us to provide this highly innovative therapy to babies born with MoCD Type A, a severely progressive genetic disease that results in irreversible neurological damage and eventual death," said Meir Jakobsohn, Founder and CEO of Medison Pharma. "The Israeli Ministry of Health's decision brings us one step closer to accelerating access of this cutting-edge therapy to those in need."
In March 2022, Sentynl acquired the global rights to NULIBRY and is responsible for the ongoing development and commercialization of NULIBRY in the U.S. and developing, manufacturing, and commercializing fosdenopterin globally. Sentynl and BridgeBio share development responsibilities through the approval of the marketing authorization application under accelerated assessment with the European Medicines Agency.
