Molybdenum cofactor

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 autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity.1,2 The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.2 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 NULIBRY (fosdenopterin) for Injection is a substrate replacement therapy that provides a synthetic source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. NULIBRY was approved by the U.S. FDA in February 2021, and by the Israel Ministry of Health in July 2022 and is indicated to reduce the risk of mortality in patients with MoCD Type A. NULIBRY was also approved by the EMA in September 2022, with an indication for treatment of patients with MoCD Type A. References 1 Mechler K et al. Genet Med. 2015;17(12):965-970. 2 Schwarz G. Cur Op in Che Bio. 2016;31:179-187. 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 , or call 1-800-FDA-1088. About Sentynl Therapeutics Sentynl Therapeutics is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl's experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and well-differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations and industry guidelines. For more information, visit . About Zydus Group Zydus Lifesciences Ltd. with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global lifesciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group has a significant presence in cancer related therapies and offers a wide range of solutions with cytotoxic, supportive & targeted drugs. The group employs nearly 25,000 people worldwide, including 1,400 scientists engaged in R & D, and is driven by its mission to unlock new possibilities in lifesciences through quality healthcare solutions that impact lives. The group aspires to transform lives through path-breaking discoveries. For more information, visit .

PALO ALTO, Calif. and SOLANA BEACH, Calif. and PETACH TIKVA, Israel, Aug. 10, 2022 /PRNewswire/ -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), 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. Nulibry® (fosdenoptrin) Understanding MoCD Type A "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. About Molybdenum Cofactor Deficiency (MoCD) Type A MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity.1,2 The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.2 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 NULIBRY®(fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group. References 1 Mechler K et al. Genet Med. 2015;17(12):965-970. 2 Schwarz G. Cur Op in Che Bio. 2016;31:179-187. About BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter. About Sentynl Therapeutics Sentynl Therapeutics is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl's experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and highly differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations, and industry guidelines. For more information, visit www.sentynl.com and follow us on LinkedIn. About Zydus The Zydus Group, with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global pharmaceutical company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group employs over 23000 people worldwide and is driven by its mission to unlock new possibilities in life-sciences through quality healthcare solutions that impact lives. The group aspires to become a global life-sciences company transforming lives through pathbreaking discoveries. For more information, visit https://www.zyduslife.com/zyduslife/. About Medison Pharma Medison is a global pharma company focused on providing access to highly innovative therapies to patients in international markets. Medison is the first to create an international commercialization platform for highly innovative therapies, helping to save and improve lives by making the best available novel treatments accessible to patients in international markets. Medison has a track record of multi-territorial partnerships with leading pharmaceutical and biotech companies seeking to expand their global reach. Medison is also an active investor in disruptive healthcare technologies and provides its partners with exposure to innovation in biotech and digital health. To learn more visit www.medisonpharma.com.