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IntraBio Receives Tay-Sachs and Sandhoff Rare Pediatric Disease Designation from the FDA

Wednesday, 12 December 2018 07:00 AM

IntraBio Inc.

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OXFORD, UK / ACCESSWIRE / December 12, 2018 / IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for rare ("orphan") and common neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation (RPDD) to its lead compound series (IB1000s) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff diseases), a rare lysosomal storage disorder that is characterized by progressive neurodegeneration and premature death.

IntraBio has previously been granted Orphan Medicinal Drug Designation from the European Commission and Orphan Drug Designation from the FDA for IB1000s for the treatment of GM2 Gangliosidosis.

Sue Kahn, Executive Director of the National Tay-Sachs & Allied Diseases Association (NTSAD), commented on IntraBio's RPDD for GM2 Gangliosidosis, "On behalf of all children and adults who suffer from Tay-Sachs or Sandhoff disease, National Tay-Sachs & Allied Diseases Association looks forward to the day when there will be approved treatments that will meaningfully improve their quality of life."

The Rare Pediatric Disease Designation makes IB1000s eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of New Drug Authorization (NDA). This PRV can be redeemed to receive a priority review of a subsequent marketing application for a different product and can be sold or transferred an unlimited number of times. In 2016, four vouchers were sold for an average price of $200 million each.

"The value that accompanies this Rare Pediatric Disease Designation will greatly aid IntraBio in further developing treatments for rare, genetic diseases with high unmet medical needs," noted Professor Mallory Factor, Chairman of IntraBio. "There are currently over six thousand rare, genetic diseases that have no available treatments, and this designation will help provide IntraBio with additional resources to develop novel therapies for patients with these debilitating, often fatal conditions."

GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and are caused by mutations in the HEXA gene, which disrupts the activity of the enzyme beta-hexosaminidase A, preventing the enzyme from breaking down GM2 gangliosides. As a result, GM2 gangliosides accumulate to toxic levels, particularly in neurons in the brain and spinal cord, leading to cell death and resulting in the signs and symptoms of Tay-Sachs and Sandhoff disease. There is nothing medically available for the treatment of GM2 Gangliosidosis at this time.

IntraBio, with its collaborators, has evaluated the effect of IB1000s in compassionate-use studies in over 175 patients, forming the scientific basis for IB1000s to be further investigated for the treatment of 18 indications, including neurodegenerative diseases and lysosomal storage disorders. Future opportunities to develop the IB1000s series further in additional indications include Lewy Body Dementia (LBD), Parkinsonisms, Restless Leg Syndrome (RLS), Amyotrophic Lateral Sclerosis (ALS), and Multiple Sclerosis (MS), all of which of have high-unmet medical needs.

IntraBio is currently in the process of applying for multi-national clinical trials with its lead asset (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease), Niemann-Pick disease Type C (NPC), and certain inherited Cerebellar Ataxias (CA).

About IntraBio

IntraBio Inc. is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases. IntraBio's platform results from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders from the University of Oxford and the University of Munich.

IntraBio's management team and consultants have vast commercial experience and a successful track record of drug development in the USA and Europe. Together, IntraBio's team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.

IntraBio Inc. is a US corporation with its principal laboratories and offices in Oxford, United Kingdom.

For further information please contact:

Cass Fields

[email protected]

www.intrabio.com

SOURCE: IntraBio Inc.

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