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Print Edition StoriesThursday, March 3, 2011
Infinity Pharma gets FDA orphan drug status
By Lori Valigra, Mass High Tech correspondent
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Infinity Pharmaceuticals Inc. of Cambridge said the U.S. Food and Drug Administration has granted orphan drug status to its IPI-926 drug candidate to treat chondrosarcoma, and that it has started another Phase 2 trial.
Chondrosarcoma is a rare cancer that results in abnormal bone or cartilage growth. It affects about 2,000 Americans each year, according to the Mayo Clinic, often in adults aged 50 to 70. The new Phase 2 trial is designed to evaluate the safety and efficacy of IPI-926 compared to placebo in patients with metastatic or locally advanced, inoperable chondrosarcoma.
The trial is expected to enroll more than 100 patients worldwide. The company said there are no approved systemic treatments for the disease and when surgery is no longer possible, it is fatal.
IPI-926 is an oral drug candidate that inhibits Smoothened, a key component of the Hedgehog pathway. In chondrosarcoma, the Hedgehog pathway is believed to be involved in tumor initiation, growth, survival and spread. Inhibiting Smoothened with IPI-926 may treat chondrosarcoma by disrupting the Hedgehog pathway and inhibiting tumor growth.
“There is a strong preclinical rationale for treating chondrosarcoma by inhibiting the Hedgehog pathway with IPI-926,” Julian Adams, president of research and development at Infinity, said in a statement.
Orphan drug status encourages companies to develop compounds for rare diseases as they may receive funding for certain clinical trials, clinical trial design assistance, tax credits, a waiver of Prescription Drug User Fee Act fees and marketing exclusivity for up to seven years following regulatory approval.
The company also recently announced that it will be presenting at the Cowen and Company 31st Annual Health Care Conference in Boston on March 7 and at the Barclays Capital 2011 Global Healthcare Conference in Miami on March 16.
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