Viken – 28th August 2020 – TIKOMED, a Swedish privately-held biopharmaceutical company today announced that the European Commission has granted Orphan Drug Designation to its lead drug candidate ILB® for the treatment of patients with amyotrophic lateral sclerosis (ALS) – a terminal, neurodegenerative disease where most patients die of respiratory muscle weakness in less than three to five years from symptom onset and for which there is yet no cure.
Orphan Drug Designation (ODD) in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). ODD for ILB® provides significant economic, scientific and regulatory benefits, including reduced fees and taxes, access to free protocol assistance and scientific advice from the EMA, access to a centralized market authorization procedure, and, if approved, 10 years of EU market exclusivity.
“With the approval from The Food and Drug Administration (FDA) already in place earlier this summer, this designation from the Committee for Orphan Medicinal Products further acknowledges the compelling clinical data with ILB® from our ongoing clinical trial program in ALS”, said Anders Kristensson, CEO of TIKOMED. “This orphan drug designation for our lead drug candidate ILB® for the treatment of ALS, is a significant milestone for us in our mission to develop a promising new therapeutic treatment for this chronically debilitating and life-threatening disease for which there is a great unmet need.
To qualify for orphan drug designation, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes.
Following the Commission’s decision in May 2020, to grant TIKOMED Small and Medium-Sized Enterprise (SME) status, TIKOMED is currently preparing for a scientific advisory meeting with the EMA to evaluate and speed up the development plans for ILB®
Amyotrophic lateral sclerosis (ALS) is a lethal progressive disease that causes degeneration of motor neurons (nerve cells that control muscle cells) and muscle. As motor neurons are lost, the muscles they control become weak and non-functional, thus leading to muscle weakness, disability, and eventually death. The clinical signs of ALS are weakness and atrophy of voluntary muscles, increased muscular tone with increasing spasticity and decreased fine motor skills, as well as increasing difficulties of swallowing, speech and respiration. From the time of diagnosis, most patients die within three to five years. No treatment has previously been found to halt or reverse the progress of the disease and even slowing disease progression evades many patients.
Tikomed’s drug candidate, ILB® is a patented Dextran Sulphate which addresses the underlying causes of neurodegeneration in acute and chronic diseases. ILB® targets multiple cellular pathways responsible for the loss of neuron structure or function seen in the neurodegenerative disease processes, boosting self-repair and protection, enabling neurons to regain lost functions.
Tikomed AB is a privately, owned company based in Viken, Sweden. Tikomed is developing therapeutics for treating acute and degenerative neurological and ophthalmic diseases (ILB® program) and an infusion product for improving outcomes in cell therapies (IBSOLVMIR® program).