Pfizer Opens Access To EAP Program For Breast Cancer Drug

Pfizer announced that it making its investigational drug Palbociclib available under an expanded access program (EAP) in the U.S.

Palbociclib is an investigational CDK 4/6 inhibitor that works to restore normal cell cycle control and stop the spread of tumor cells. The drug is under assessment as a potential new treatment for patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer. These represent the largest subgroup of women with the disease. The company recently submitted a New Drug Application (NDA) for palbociclib with the FDA.

The EAP program will be a multi center, open label, single-arm study open to post-menopausal women with HR+, HER2- advanced breast cancer. Participants will be treated with palbociclib in combination with letrozole.

Dr. Mace Rothenberg, SVP of Clinical Development and Medical Affairs, CMO of Pfizer Oncology, said, “With recruitment of new patients to our Phase 3 PALOMA-2 and PALOMA-3 trials now complete, Pfizer is initiating the Palbociclib Expanded Access Program. This program will provide a mechanism by which eligible women who may benefit from treatment with palbociclib can gain access to this investigational therapy at this time.”

The FDA defines expanded access as the use of an investigational drug outside clinical trials to treat a patient with no other treatment option for their serious or immediately life-threatening disease or condition. Also known as ‘compassionate use,’ EAP was preceded by an inflexible case-by-case basis of investigational drug use in the mid-1990s. EAP has since been expanded to allow greater flexibility and availability for patients in need. However, some caution that EAP may be abused to the detriment of patients that seek to benefit from unapproved, investigational drugs. This year’s highly publicized story of Josh Hardy and Chimerix’s investigational anti-viral drug brincidofovir is pushing lawmakers, regulatory officials, and drug makers alike to take another look at the need for new guiding principles for EAP.