The U.S. Food and Drug Administration (FDA) has granted orphan drug status to combinations of VTS-Aspirin plus fumarate therapy using Vumerity (diroximel fumarate) or Bafiertam (monomethyl fumarate) for easing flush in patients with relapsing-remitting multiple sclerosis (RRMS).
In the U.S., orphan drug designation is given to medicines with potential to be safe and effective treatments for rare conditions affecting no more than 200,000 people. It provides regulatory support and financial benefits to help accelerate the clinical development of these VTS-Aspirin combinations, and guarantees a seven-year period of marketing exclusivity if granted regulatory approval.
The recently granted status follows another orphan drug designation given to Vitalis‘ lead candidate VTS-72, a proprietary formulation of dimethyl fumarate (marketed as Tecfidera by Biogen) plus VTS-aspirin for RRMS patients who experience flushing after taking Tecfidera.
“Because of the potential clinical benefit of combining these fumarates with aspirin, we were able to obtain Orphan Drug Status for each, in combination with VTS-Aspirin, to treat patients that experience fumarate flush,” Joseph Habboushe, MD, MBA, founder of Vitalis, said in a press release.
“We believe that FDA’s recognition of this subset of patients as an orphan population speaks to the unmet need for an effective method of treating fumarate flush, as our product candidates are the first adult MS therapies to receive orphan designation in over 15 years,” Habboushe added.
Tecfidera is one of the most prescribed oral therapies to prevent relapses and slow disease progression in RRMS patients. But those taking the therapy often complain of flushing, a troublesome side effect characterized by feelings of warmth and a rapid reddening of the skin.
This side effect, which affects quality of life and can cause patients to discontinue treatment, is also seen with the more recent treatments, Vumerity (also marketed by Biogen) and Bafiertam (by Banner Life Sciences), whose active substance fumarate is the same as that of Tecfidera.
“Fumarates, an important class of oral drugs to treat RRMS, cause fumarate flush in as many as 40% of patients. This is a significant side effect and can lead to skipping of doses or, in some cases, treatment discontinuation,” Habboushe said.
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