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.
Aspirin has been used for decades to reduce the side effects of fumarate-induced flushing. But adherence to aspirin as a treatment is low, since patients need to take it at least 30 minutes before the fumarate treatment for it to have an effect.
Vitalis’ researchers developed a novel formulation of aspirin, called VTS-Aspirin, that can be delivered at the same as the fumarate treatment, and thus could be combined with approved therapies or into one single medication, such as VTS-72.
The combination of VTS-Aspirin and dimethyl fumarate in VTS-72 was investigated in a randomized clinical trial in 18 healthy people, where it reduced flushing by 63.3% compared with standard Tecfidera. It also caused no flush in more than one-third of participants who had experienced flushing while on Tecfidera.
Vitalis is planning to launch a Phase 3 trial before the end of the year, which is expected to provide data for VTS-72’s potential approval as a treatment of fumarate-related flush in RRMS.
“We believe that combining fumarates with VTS-Aspirin can bring meaningful tolerability benefits to RRMS patients, and we will continue to evaluate the potential of these combinations,” Habboushe concluded.
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