Inceptua Early Access and Sentynl Therapeutics Inc., a U.S.-based biopharmaceutical company bringing therapies to patients living with rare diseases, have entered into a partnership.
The partnership is to make Sentynl’s Nulibry available via an early access program for eligible patients with molybdenum cofactor deficiency (MoCD) Type A.
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 four years of life if left untreated.
Nulibry is the first approved treatment option for MoCD Type A. It was approved by the U.S. Food and Drug Administration (FDA) in 2021, and in September 2022 the European Commission granted marketing authorization for Nulibry under exceptional circumstances.
Early Access Programs (also known as expanded access or early access) are a route through which medicines that 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.
Critical treatment for children
“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.
Some side-effects of Nulibry include oversensitivity 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.
Grant Castor, senior vice president 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.”