FDA Grants Orphan Drug Status to Livantra, Repurposed for Treatment of PAH

The U.S. Food and Drug Administration has granted orphan drug designation to Livantra, a new formulation of an already approved treatment outside the U.S. for another indication, for the treatment of pulmonary arterial hypertension (PAH), according to the drug’s developer, Martin Pharmaceuticals.

In March this year, Livantra also received FDA orphan drug status  for the treatment of another condition, acute or chronic liver failure.

The FDA’s Orphan Drug Designation aims to encourage and speed the development of treatments for rare diseases,  those affecting fewer than 200,000 patients in the U.S., which are typically overlooked by pharmaceutical companies.

The status offers tax benefits for clinical research costs and grants the drug developer a seven-year licence for exclusive marketing in the U.S., if the drug is approved.

The therapy is a new liquid formulation of trimetazidine (brand name Vastarel, among others), a medication currently approved in Europe and other countries for the treatment of angina pectoris. In the U.S., however, the medication has never been approved for any indication.

Trimetazidine is an anti-ischemic agent, a compound that counters the effects of constraints in the blood supply and the shortage of oxygen in tissues, usually caused by vascular malfunctions such as angina.

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Considering that PAH also is a vascular condition, marked by the obstruction of lung arteries, Martin Pharmaceuticals is hoping the FDA approves trimetazidine as a repurposed drug to be used in the treatment of this condition, as well.

Livantra restores the activity of an enzyme called pyruvate dehydrogenase, which has low levels in PAH. By doing so, the treatment induces a shift in the metabolism of sugar (or glucose) which prevents excessive cell growth proliferation inside blood vessels.

Livantra’s potential for treating PAH has been supported by preclinical studies, Martin Pharmaceuticals claims. In two mouse models of PAH, the drug was seen to significantly decrease pulmonary arterial pressure, reduced right heart ventricle (lower heart chamber) thickness, and improve exercise capacity.

Martin Pharmaceuticals’ pipeline focuses on finding new indications for therapies that have already been approved, or repurposed drugs, a strategy that increases the chances of patients seeing a new therapy sooner.

Because an approved medication has already been tested for safety and effectiveness, this strategy for repurposed drugs reduces the time, cost, and risk of clinical trial failures, and provides the means for safer and more effective treatments to become available.

In the case of Livantra, its active ingredient, trimetazidine, has already been proven to be safe and tolerable, Martin Pharmaceutical states.