Tag Archives: global orphan cancer drug

TiGenix’s Cx601 granted as orphan drug designation in Switzerland

TiGenix NV, a biopharmaceutical company, focused on developing novel therapeutics from its proprietary platform of allogeneic expanded stem cells, declared that its leading product Cx601, has been granted orphan drug designation status in Switzerland, for the treatment of complex perianal fistulas in Crohn’s disease patients. Cx601 is developed by TiGenix and licensed to Takeda Pharmaceutical Company Limited for development and commercialization outside the US.

Cx601 is the suspension of allogeneic adipose-derived stem cells (eASC) injected within a lesion for curing complex perianal fistulas in patients with Crohn’s disease which is irresponsive to at least one of the conventional or biologic treatment. Crohn’s disease is the inflammatory disease of intestines. Patients suffering with crohn’s disease develop complex perianal fistulas for which there not treatments options available.

Swiss agency declared orphan drug status for therapeutic products regarding the application dossier for Cx601, or eASCs, adipose-derived stem cells for the rare disease anal fistulas. This was based on the orphan drug designation by European commission in 2009, which defines proof of quality, efficacy and safety and rarity of the disease as well.

Biologics and medical treatments are designated as orphan drug being investigated for the treatment of rare diseases and medical conditions. In Europe, rare diseases can be defined as the disease affecting fewer than 5 out of 10,000 people.



For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

EMA announces orphan drug designation for ABO-102 gene therapy

Abeona Therapeutics Inc., a biopharmaceutical company dedicated to develop gene therapies for life threatening rare diseases declared that leading therapy ABO-102 has been designated as orphan drug b European Medicines Agency (EMA) for the treatment of Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease causing neurocognitive decline, speech of loss, premature death in children and loss of mobility.

Abeona’s MPS IIIA program has been previously granted the designation of orphan drug by Food and Drug Association (FDA) in the US and received the Rare Pediatric Disease Designation as a pre-requisite part of the Priority Review Voucher (PRV) process.

The FDA and EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan Status can be applied to products proven safe and effective in treating conditions that affect a relatively small number of patients, and the designation may entitle up to seven years of US marketing exclusivity upon regulatory approval. Companies whose drugs have an orphan designation also receive certain tax credits and are exempted from paying prescription drug user fees normally required of companies submitting products for approval.



For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA grants BioXcel’s BXCL101 orphan drug designation for Neurofibromatosis Type 2 (NF2)

BioXcel, a private pharmaceutical company in Connecticut, declares tht FDA has granted BXCL101, orphan drug designation status for the treatment of the patiets suffering from Neurofibromatosis Type 2, disease associated with neurologic and ophthalmologic abnormalities caused by benign tumors of the brain, spinal cord and peripheral nerves.

BXCL101 is a proprietary version of an approved drug, bortezomib, adapted for chronic use in NF2 patients with both a novel dosing regimen and delivery approach.  NF2 is the first drug discovered on BioXel’s R&D platform to be granted orphan drug designation.

Under the U.S. Orphan Drug Act, the FDA provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S.  This designation makes BXCL101 eligible for a seven year marketing exclusivity period.  In addition, it provides certain incentives, including federal grants, prioritized consultation by the FDA on clinical studies, tax credits and a waiver of PDUFA filing fees.  The orphan designation does not amend the standard regulatory requirements, timing and process for obtaining marketing approval.



For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com