Tarrytown Firm Receives Orphan Status for Drug

A new drug that may help people suffering from a painful ailment has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The drug is NP-1 and it is being developed by EpiCept Corporation of Tarrytown.

EpiCept Corporation develops and commercializes drugs for the treatment of cancer and pain. The firm does not conduct research, but instead licenses products from other companies and institutions, said Robert Cook, senior vice president and chief financial officer at EpiCept. The company has been in Tarrytown since 2007.

Granting orphan drug status is one way the federal government promotes the development of treatments for orphan diseases, which are those diseases that affect fewer than 200,000 people. Pharmaceutical companies may not be able to recoup their investment in a treatment for an orphan disease because of their rarity, which can limit research into treatment options. Pharmaceutical firms receive economic incentives from the government to develop orphan drugs if the drug and disease meet certain criteria.

"Getting orphan drug means that we will market exclusivity for seven years after approval," Cook said. This exclusive right to market NP-1 in the United States is regardless of when the patent on the drug runs out, he added.

EpiCept licensed NP-1 from Dalhousie University in Nova Scotia, Cook said, and the company has global rights to the drug. Phase III studies—the last stage of clinical trials before an application for approval is submitted to the FDA—are still to be done. If all goes well, these studies will take about three years, with approval perhaps another year after that, he noted.

NP-1, which is a skin cream, was given orphan drug status as a treatment for a condition called post-herpetic neuralgia, which can occur in some people who develop shingles. Shingles is a very painful blistering rash caused by the same Herpes Zoster virus that causes chicken pox. The virus can stay dormant in the body for years after someone contracts chicken pox, but then emerge as the painful rash of shingles. Although shingles usually lasts a few weeks, for some people, the severe pain lingers for years after the rash has cleared up.

"Shingles is bad, but post-herpetic neuralgia is a chronic condition. People have the pain for the rest of their lives," Cook said. While shingles is not uncommon, post-herpetic neuralgia only happens in a few cases of shingles and is an orphan disease, he added.

Post-herpetic neuralgia is one form of peripheral neuropathy, which is pain caused by damage to nerves outside of the brain and spinal column. Other causes of peripheral neuropathy include diabetes, liver failure, and the side effects of radiation therapy and chemotherapy for cancer. EpiCept is also studying NP-1 as a treatment for neuropathy caused by chemotherapy.

The active ingredients in NP-1 are amitryptyline and ketamine, both of which already have FDA approval for other uses. Their formulation as a topical cream to be used on the skin is patented.

EpiCept also makes Ceplene, which has been approved in Europe for maintaining remission of acute myeloid leukemia. The company is also developing two cancer drugs that have been shown to disrupt the blood vessels in solid tumors.