Immune Pharmaceuticals, Inc., a biopharmaceutical company developing novel therapeutic agents for the treatment of immunologic and inflammatory diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bertilimumab for the treatment of bullous pemphigoid (BP).
“I am truly grateful that the FDA has granted Fast Track designation to bertilimumab for the treatment of bullous pemphigoid. This important achievement follows the recent granting of Orphan Drug Designation in both the United States and Europe, which together demonstrate the regulatory affairs momentum our team has achieved,” commented Immune’s Interim Chief Executive Officer, Tony Fiorino, MD, PhD. “There is no doubt that bertilimumab development will benefit from the opportunity to have more frequent contact with the FDA, particularly now, as we move forward with a new manufacturing process and plan for a phase 2/3 pivotal study in bullous pemphigoid.”
The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions that are unmet medical needs. Fast Track designation enables more frequent interactions with the FDA in order to shorten the development and review process, and may include potential eligibility for Accelerated Approval, Priority Review and Rolling Review.
Immune Pharmaceuticals, Inc., a biopharmaceutical company developing novel therapeutic agents for the treatment of immunologic and inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to bertilimumab for the treatment of bullous pemphigoid (BP).
“We are incredibly gratified that bertilimumab has received Orphan Drug Designation for the treatment of bullous pemphigoid from the FDA and believe this designation, coupled with the recent positive opinion from the [European Medicines Agency] EMA’s Committee for Orphan Medicinal Products represent a significant regulatory milestone or bertilimumab,” commented Immune’s Chief Medical and Operating Officer, Tony Fiorino, MD, PhD. “We are focused on putting all of the manufacturing and regulatory pieces in place to launch a pivotal phase 2/3 study of bertilimumab in bullous pemphigoid next year.”
The FDA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that affect fewer than 200,000 people in the U.S. This designation provides for a seven-year marketing exclusivity period, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.
Chances are you have wondered why there are not more treatments available for pemphigus and pemphigoid, or at minimum, why there is not more research underway for these conditions. There are a few reasons for this, and unfortunately, it’s something that is not easily solved.
First and foremost, pemphigus and pemphigoid represent a rare group of conditions. A rare disease is also referred to as an orphan disease, and is defined as one that affects less than 200,000 Americans. Drug companies typically spend money on research and development for diseases that affect a larger group of people. In 1983, the Orphan Drug Act was passed. This Act incentivizes research for treatments of rare diseases. The three main incentives include federal funding grants to initiate clinical trials, tax credits of up to 50% and an exclusive right to market drugs for a period of seven years from FDA-approval date. Since 1983, many new medications were approved for existing diseases, and many existing medications were approved for new indications.
Studies on rare conditions like pemphigus and pemphigoid are challenging to get participation and potential subsequent FDA-approval for treatments for these conditions. P/P are rare, but additionally, most people with these conditions typically have had difficulties getting properly diagnosed and then the effective treatment for that diagnosis. When placebo is involved it means the chance of going without treatment which means the potential for relapse. Although rescue protocols for this are in place, it can still be scary to some people. Other challenges to recruitment into orphan drug trials include poor disease awareness and a small number of physicians who actually treat the condition. There also may be inclusion even when there is interest because people may have been treated already in a way that excludes them.
The IPPF has done a lot to try to work toward better enrollment in clinical trials by raising awareness among the specialists that would see people who have pemphigus and pemphigoid, and keeping a database of physicians who have at any time treated these conditions. As of October 20, 2015, there are 31 clinical trials in various phases for pemphigus and 30 for pemphigoid. Some involve non-drug research, others are for existing drugs to be approved for this indication and others are for drugs in development.
If you are interested in knowing more about these trials or would like to explore enrolling, talk with your physician and discuss this. You can also get more info and contact information on www.clinicaltrials.gov and take a look at the inclusion criteria and the participating centers.