Rare Disease Report

Rigel CEO Encouraged by Company's Progress and Recent FDA Activity

DECEMBER 12, 2017
Raul Rodriguez
Rigel Pharmaceuticals is at the NDA Review of its lead product Fostamatinib, intended to treat immune thrombocytopenia. In 2018, the company has plans to pursue a second indication. President and CEO Raul Rodriguez is encouraged by his company's success and the FDA's recent activity focused on rare hematologic disease.

Rare Disease Report caught up with Rodriguez at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition last week, where he spoke about how excited he is for the company's future as it heads into 2018.

 

Rodriguez: We are at the NDA Review for our lead product, Fostamatinib, for the treatment of immune thrombocytopenia - ITP - which is a rare autoimmune disease where the body destroys its own platelets. These people have very low platelet counts, and as a result, they have difficulty clotting. They have exposure for tremendous bleeding episodes, some minor, but many of them also major bleeding episodes; it’s actually quite a severe disease. So, it’s an exciting time.

We have a new drug potentially approved next year for the treatment of that disease, so I’d love to tell you more about it. It’s a small patient population so recruitment of clinical trials is really very difficult sometimes. We had to go in countries far field in order to recruit the trials - not a big trials. We did two trials of 75, 150 patients in total. Nonetheless, that took us close to two years to enroll, and we did them in the U.S., Canada, various countries in Europe, Australia. But it really required going to all those countries in order to find the right patients.

Our second indication, also a rare disease, is autoimmune hemolytic anemia. Autoimmune hemolytic anemia, as the name implies, is an autoimmune disease where the body destroys its red blood cells and these people are highly anemic and have very difficult lives. We just completed the first part of a Phase 2 trial and we’re going to have discussion with the FDA on how best to proceed towards an approvable path indication as well, so we will be able to disclose that information. Easy to say the next 6 months, 7 months will be a very exciting time for us. I think it’s really refreshing to see and rewarding, I think, for patients to recognize that even patients who have limited numbers set from a particular disseize are now getting substantial attention and it’s great to see the FDA standing behind that. In fact, encouraging those patients to give them feedback, I think that’s very useful as well. It’s really been a monumental change, I think, for those patients suffering from rare disorders and I think we’re a very key example of that where the FDA has been willing to work with us.

We have orphan designation for our lead indication and are going to apply for autoimmune hemolytic anemia and it’s been a tremendous help being able to work with the agency in terms of their review of that opportunity and also to engage with the patient suffering from this disease and the patient advocacy organization, the PDSA in this case, being really active in advocating for their patient population. So it’s really been a wonderful exchange where now we can focus on these rare diseases not just the big diseases with many many millions of patients, perhaps.

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