FDA grants orphan drug designation to Algernon’s ifenprodil, while exclusivity remains unclear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Ifenprodil, also known as NP-120, is an N-methyl-D-aspartate (NMDA) receptor antagonist. NMDA receptors are found on many tissues, including lung cells, and are targets among painkillers and anesthetics. However, Algernon touts ifenprodil as a potential first-in-class option for IPF and chronic cough.

According to updated Phase IIa data released back in July, ifenprodil helped reduce patients’ mean 24-hour cough counts by 32% at 4 weeks (p = 0.023) and 39.5% at 12 weeks (p = 0.001) compared to baseline.

Christopher Moreau

“We appreciate the U.S. FDA’s decision to grant ODD status to Ifenprodil for IPF, a disease for which prognosis remains dismal, with 50% mortality expected within 3-4 years,” Algernon CEO Christopher Moreau said in a news release.

The news comes as the FDA is “not currently finalizing ODE [orphan drug exclusivity] determinations” because of the “far-reaching implications” of a recent court case involving Catalyst and Jacobus. A US appeals court overturned a prior FDA court win, ruling that the agency shouldn’t have approved a rare disease drug because a previously approved (and more expensive) one with the same ingredient had orphan drug exclusivity.

The FDA told Endpoints News in a statement last week:

The decision has caused uncertainty for rare disease drug development. Under the court’s interpretation of the scope of ODE, ODE would block approval of another company’s application for the same drug for the entire disease or condition for which the drug is granted orphan-drug designation, regardless of whether the drug was approved only for a narrower use or indication. Under that interpretation, a sponsor could seek approval and exclusivity for a drug by focusing on the smallest, easiest-to-study populations and such exclusivity would block the drug for the entire disease, even though the sponsor did not invest in studying and developing the drug for all individuals with the disease.

The agency told Endpoints News on Monday that it had no updates to provide.

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