MMRF joins multiple myeloma community in celebrating FDA accelerated approval of Pomalyst

Feb 10 2013

The Multiple Myeloma Research Foundation (MMRF) today joined the multiple myeloma community in celebrating the U.S. Food and Drug Administration's (FDA) decision to grant accelerated approval of Pomalyst^® (Celgene Corporation, pomalidomide) for the treatment of patients with relapsed and refractory multiple myeloma who no longer respond to currently available treatments. A critical advance for the treatment of this incurable blood cancer, together with Kyprolis^TM (carfilzomib), Pomalyst marks the second treatment to receive accelerated approval in the last seven months, which is unparalleled in any other cancer.

"Today marks another historic day - the availability of an unprecedented second breakthrough therapy for this fatal disease within just seven months for patients who have run out of treatment options," stated Kathy Giusti, Founder and CEO of the MMRF and the Multiple Myeloma Research Consortium (MMRC), and a multiple myeloma patient. "We are grateful for the FDA's decision and for the unwavering commitment of our partner Celgene to the multiple myeloma patient community, and are proud to have accelerated the development of this promising new treatment."

In 2008, the MMRC entered into collaboration with Celgene and bolstered the clinical development of Pomalyst. Since that time, the MMRC:

• Facilitated the Phase I and Phase II clinical trials that provided the basis for the accelerated approval, which included assistance with protocol development and Institutional Review Board (IRB) submissions.
• Drove rapid patient enrollment to the Phase I and Phase II registration trials, enrolling 100% of patients in the Phase I trial and more than 80% in the Phase II trial, completing enrollment in less than two years and exceeding timeline expectations by several months.
• Provided dedicated site management and guidance on key safety and efficacy issues throughout the Phase I and Phase II registration trials.
• Supported further investigation of Pomalyst for multiple myeloma; to date, the MMRC has opened four trials involving pomalidomide, two of which are novel combinations that are currently open for enrollment.

Pomalyst was approved by the FDA for patients who have received at least two prior therapies including Revlimid^® (lenalidomide) and Velcade^® (bortezomib) and have demonstrated disease progression on or within 60 days of completion of the last therapy, and will only be available in the United States through POMALYST REMS™, a restricted distribution program.

Source: Multiple Myeloma Research Foundation (MMRF)