Sentynl Therapeutics Announces Continued Availability of NULIBRY® (fosdenopterin) and the Launch of Sentynl Cares™ Patient Access Program.

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Apr 25, 2022, 09:00 ET

SOLANA BEACH, Calif., April 25, 2022 /PRNewswire/ -- 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.), today announced the continued availability of NULIBRY (fosdenopterin) for Injection and the launch of Sentynl Cares. NULIBRY is approved by the U.S. Food and Drug Administration (FDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening pediatric genetic disorder. Sentynl Cares provides a comprehensive set of support programs dedicated to helping children with MoCD Type A in the United States gain access to NULIBRY.

Recognizing that most patients with MoCD Type A experience delayed or missed diagnoses that may affect patient outcomes, Sentynl plans to advance initiatives aimed at providing an earlier diagnosis and treatment of the disease.

These initiatives include:

Supporting the development of a novel newborn screening test, including facilitating the collection of whole blood samples from patients with MoCD Type A disease for assay development and validation, with the aim of universal screening for all babies in the first days of life.
Advocating for the inclusion of the MOCS1 gene to the commercially available genetic test panels that will aid in the diagnosis of MoCD Type A patients.
Launching Sentynl Cares a comprehensive set of support programs dedicated to helping children get access to our therapies. Sentynl Cares also provides tools and resources to help facilitate the access process.

Sentynl's Chief Executive Officer, Matthew Heck, said, "Early diagnosis and therapeutic intervention is critical to MoCD Type A disease patients and their caregivers. Our company is proud to advance initiatives that are aimed at earlier diagnosis and treatment of rare diseases like MoCD Type A."

Speaking on the development, Dr. Sharvil Patel, Managing Director, Zydus Lifesciences Ltd., said, "These initiatives align with our core purpose to empower patients suffering from rare disease with the freedom to live healthier and fulfilled lives. Zydus is proud that NULIBRY (fosdenopterin) is now among its portfolio of rare disease treatments designed improve outcomes for patients and their caregivers."

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 the United States are not known, but the estimated incidence is 1 per 100,000 live births.³ Based on these estimates, MoCD Type A is likely to be underdiagnosed, with an estimated 22 to 26 missed diagnoses per year in the United States and European Union.

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.

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION

NULIBRY is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.

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%), and cough/sneezing (44%). 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 Cares:

Sentynl Cares is a comprehensive set of support programs dedicated to helping children get access to our therapies. Sentynl Cares also provides tools and resources to help facilitate the access process.

References
¹Mechler K et al. Genet Med. 2015;17(12):965-970.
²Schwarz G. Cur Op in Che Bio. 2016;31:179-187.
³Mayr SJ, et al. Forecasting the incidence of rare diseases: an iterative computational and biochemical approach in molybdenum cofactor deficiency type A. Presented at the 2019 SSIEM meeting; September 3-6, 2019; Rotterdam, The Netherlands.

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 highly 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.

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 path breaking discoveries. For more information, visit https://www.zyduslife.com/zyduslife/.

Sentynl Therapeutics Contact:
Michael Hercz
[email protected]
888-507-5296

SOURCE Sentynl Therapeutics