FDA Grants Orphan Drug Designation to Pancreatic Cancer Treatment

Biopharmaceutical company BERG announced this morning that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its leading product candidate BPM31510 (ubidecarenone) for the treatment of pancreatic cancer.

The potential therapy is a first-in-class molecule being developed to specifically target the dysregulated metabolism commonly observed in cancer.

Because less than 200,000 people in the U.S. have been diagnosed with metastatic pancreatic, qualifying it as rare, it is still the 4th leading cause of all cancer-related deaths in the country. Per the National Cancer Institute, approximately 46,000 new cases of pancreatic cancer in the United States are diagnosed each year, and about 40,000 patient deaths.

Symptoms of pancreatic cancer don’t present until well after the disease has progressed, which often results in a delayed diagnosis and difficulty as it pertains to treatment.

A phase 1 clinical trial established the safety of BPM31510, and BERG has since initiated a Precision Medicine-driven phase 2 clinical trial design for the drug alone and in combination to evaluate the efficacy in patients with advanced pancreatic cancer in the U.S. and Europe.

"The FDA's orphan-drug designation marks an important milestone for BERG," said Niven R. Narain, BERG President and Chief Executive Officer in a press release. "We look forward to working closely with the FDA and other regulatory authorities to advance the clinical development of this therapeutic and map out the most effective match for treatment of pancreatic cancer.”

With the Phase 2, BERG is hoping to identify and validate molecular profiles representative of BPM31510 mediated outcomes, which could potentially lead to a diagnostic panel for patient stratification.

In May, Rare Disease Report spoke with Niven R. Narain, CEO of BERG, about a partnership between the company and the Dytrophic Epidermolysis Bullosa Research Association of America (debra of America). The two sides are coordinating to develop a topical cream formulation of BPM31510 to increase wound healing and serve as an anti-inflammatory for patients with epidermolysis bullosa (EB), a far cry from the majority of BERG’s clinical trials which are primarily focused on oncology.

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