Pfizer Receives Breakthrough Therapy Designation From FDA For PF-06651600, An Oral JAK3 Inhibitor, For The Treatment Of Patients With Alopecia Areata

- Dec 20, 2018-

Pfizer Inc. (NYSE:PFE) today announced its investigational oral Janus kinase 3 (JAK3) inhibitor PF-06651600 received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with alopecia areata, a chronic autoimmune skin disease that causes hair loss on the scalp, face, or body.1,2


The Breakthrough Therapy designation for alopecia areata was supported by positive results from a Phase 2 study, which will be presented during the late-breaking news session at the 27th European Academy of Dermatology and Venerology (EADV) Congress in Paris on September 15, 2018. Currently, there are no FDA-approved treatments for alopecia areata, which impacts millions of people worldwide and is often associated with profound psychological consequences.1,2


“We are encouraged by this Breakthrough Therapy designation as it underscores the potential of our JAK3 inhibitor to address a critical unmet need,” said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development. “We are continuing to work closely with the FDA on the development process with the goal of bringing this potential new treatment to patients living with alopecia areata as soon as possible.”


Breakthrough Therapy designation was initiated as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) signed in 2012. As defined by the FDA, a Breakthrough Therapy is a drug intended to be used alone or in combination with one or more other drugs to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug is designated as a Breakthrough Therapy, the FDA will expedite the development and review of such drug.3


Pfizer is also working with the European Medicines Agency (EMA) on the clinical development program for PF-06651600.


About Alopecia Areata

Alopecia areata is an autoimmune disease, characterized by hair loss, often patchy, on the scalp, face, or body.1,2 People suffering from alopecia areata experience symptoms when immune cells attack healthy hair follicles, causing the hair to fall out, often starting with smooth, round patches.1,2 The mean age of onset is between 25 and 35, but it can also impact children and adolescents, and is seen in both sexes and all ethnicities.1,2 More than half of patients with alopecia areata experience poor health-related quality of life and, as a result, the condition may lead to serious psychological consequences, including high levels of depression and anxiety.1


About PF-06651600 and Pfizer’s Kinase Inhibitor Leadership

The JAK pathways are believed to play an important role in inflammatory processes as they are involved in signaling for over 50 cytokines and growth factors, many of which drive immune-mediated conditions.4 JAK inhibition offers the potential for new advanced treatment options for these conditions through unique and targeted selectivity.5


“Pfizer strives to continue moving the JAK science forward with development of multiple JAKis with unique selectivity profiles that are being evaluated in clinical trials. These inhibitors have the potential of meeting significant unmet needs in multiple inflammatory conditions,” said Michael Vincent, Chief Scientific Officer, Pfizer Inflammation & Immunology.


PF-06651600 is an oral small molecule that selectively inhibits Janus kinase (JAK) 3.5 PF-06651600 is also under investigation for the treatment of rheumatoid arthritis, Crohn’s disease and ulcerative colitis.


Pfizer has established a leading kinase research capability with multiple unique kinase inhibitor therapies in development. As a pioneer in JAK science, the Company is continuing to advance several investigational programs for molecules with novel selectivity profiles, which, if approved, could potentially deliver transformative therapies for patients. In addition to PF-06651600, Pfizer has the following kinase inhibitors in trials across multiple indications:


PF-04965842: A selective JAK1 inhibitor in Phase 3 clinical trials for the treatment of atopic dermatitis(AD)6; PF-04965842 received Breakthrough Therapy designation from the FDA for the treatment of patients with moderate-to-severe AD

PF-06700841: A tyrosine kinase 2(TYK2)/JAK1 inhibitor under investigation for the treatment of psoriasis, Crohn’s disease, ulcerative colitis and alopecia areata

PF-06650833: An interleukin-1 receptor associated kinase 4 (IRAK4) inhibitor under investigation for the treatment of rheumatoid arthritis

PF-06826647: A TYK2 inhibitor under investigation for the treatment of psoriasis and inflammatory bowel disease (IBD)