On October 11, 2022 Inceptua Early Access (a business unit of the Inceptua Group), and Sentynl Therapeutics Inc. ("Sentynl"), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases reported that they have entered into a partnership to make Sentynl’s Nulibry available via an Early Access Program for eligible patients with molybdenum cofactor deficiency (MoCD) Type A (Press release, Sentynl Therapeutics, OCT 11, 2022, View Source;utm_medium=rss&utm_campaign=inceptua-early-access-and-sentynl-therapeutics-inc-announce-the-launch-of-the-early-access-program-for-nulibry-fosdenopterin-for-pediatric-patients-with-molybdenum-cofactor-deficiency-mocd [SID1234621936]).
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MoCD Type A is a rapidly progressive autosomal recessive inborn error of metabolism resulting in toxic sulfite levels causing neurologic sequelae including seizures, difficulty feeding, severe developmental delays, and death within the first 4 years of life if left untreated. Nulibry is the first approved treatment option for MoCD Type A. Nulibry was approved by the FDA in 2021, and in September 2022 the European Commission granted marketing authorisation for Nulibry under exceptional circumstances.
Early Access Programs (also known as expanded access, early access, compassionate use, named patient supply) are a compliant route through which medicines which are either still under development or not approved in their country of intended use, can be made available for patients who either have no alternative treatment option, or have exhausted all other treatment options available in their country of residence.
"Inceptua Early Access is delighted to be supporting Sentynl with the Nulibry Early Access Program to allow appropriate patients with MoCD Type A the opportunity to access the first approved therapeutic option for this disease. MoCD Type A is a devastating disease with significant unmet need and anything we can do to support early access to this new treatment option will give hope to the families of the children affected by this condition." said Stuart Bell, Vice President, Early Access, Inceptua Group.
Grant Castor, Senior Vice President, Commercial Strategy and Operations at Sentynl, said, "We are pleased to be partnering with Inceptua Early Access to provide a formal centralized process for handling requests where there are suspected and confirmed cases of MoCD Type A globally. A presumptive diagnosis of MoCD Type A and rapid initiation of treatment is critical for all children with this ultra-rare disease worldwide."