Faron Pharmaceuticals Limited (AIM:FARN) has received a major boost for its cancer drug bexmarilimab, after the US Food and Drug Administration granted it Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS).
In London, shares in the Finnish biotech ticked higher after the announcement, which could help speed up development and provide market exclusivity if the drug gets approved.
MDS is a rare blood disorder that affects fewer than 200,000 people in the US. The FDA's Orphan Drug Designation gives companies incentives such as tax breaks and exemptions from certain fees, making it easier to bring treatments for rare diseases to market.
Faron has already received the same designation for bexmarilimab in acute myeloid leukemia (AML), another aggressive blood cancer.
“Receiving FDA’s orphan drug designation for bexmarilimab for the treatment of myelodysplastic syndrome marks a significant milestone for Faron Pharmaceuticals as we continue to develop bexmarilimab for MDS and other cancers,” said Dr Petri Bono, Faron’s chief medical officer.
“This FDA ODD along with the previously granted FDA fast track designation highlights our continued progress and reinforces our belief in the potential of bexmarilimab to address this significant unmet need for treatment of MDS.”
Bexmarilimab is designed to help the immune system fight cancer by blocking a protein called Clever-1, which allows cancer cells to evade detection. By targeting this protein, Faron hopes to make existing treatments more effective. The drug is currently being tested in an ongoing clinical trial that combines it with standard treatments for MDS and AML.
The company is set to release top-line results from these trials in April, which will give a clearer picture of how well bexmarilimab is working. The company has been making steady progress in its development of the drug, with hopes that it could become a key treatment option for patients who do not respond to current therapies.
The FDA's decision strengthens Faron’s position as it continues testing the drug. If bexmarilimab proves successful in trials, the Orphan Drug Designation will provide an important advantage, including seven years of market exclusivity in the US once approved.
In early afternoon trading, the stock was up 3% at 174.4p.