Market Overview

Alector Receives Orphan Drug Designation from the U.S. FDA for AL001 for the Treatment of Frontotemporal Dementia

Share:

Alector, a privately held biotechnology company focused on developing
therapies that harness the immune system to cure neurodegenerative
diseases and cancer, today announced that the U.S. Food and Drug
Administration Office of Orphan Products Development has granted orphan
drug designation to AL001, a human recombinant monoclonal antibody, for
the treatment of all patients with frontotemporal dementia (FTD).

"There are currently no approved therapies to address the underlying
cause and needs of FTD patients. In addition, given the marked
behavioral and personality changes associated with FTD, coupled with the
early onset of the disease, the care of FTD patients is a significant
challenge for families and the society," said Robert Paul, chief medical
officer, Alector. "The granting of orphan drug designation is a
significant milestone for the AL001 development program, and we are
excited to work with leaders and experts in the research, medical and
patient advocate communities to bring AL001 to the patients suffering
from this disease."

"FTD is caused in part by a dysfunctional brain immune system,
and AL001 is one of our immuno-neurology drugs designed to repair that
immune system to elicit a therapeutic benefit. We believe that our
immuno-neurology approach could potentially have as much impact on brain
disorders as immuno-oncology drugs have had on cancer patients,"
said Arnon Rosenthal, Ph.D., chief executive officer, Alector.

About Orphan Drug Designation

The U.S. Food and Drug Administration's Orphan Drug Designation program
provides orphan status to drugs and biologics that are defined as those
intended for the safe and effective treatment, diagnosis or prevention
of rare diseases/disorders that affect fewer than 200,000 people in the
U.S., or that affect more than 200,000 persons but are not expected to
recover the costs of developing and marketing a treatment drug. Orphan
Drug Designation qualifies the sponsor of the drug candidate for various
development incentives, which may include tax credits for qualified
clinical testing, an exemption from fees under the Prescription Drug
User Fee Act (PDUFA), and a seven-year marketing exclusivity period
following approval.

About Frontotemporal Dementia

Frontotemporal dementia (FTD) is the second most common early-onset form
of dementia after Alzheimer's disease, afflicting approximately 60,000
Americans and 110,000 people in Europe. FTD is a rare but rapidly
progressing degenerative syndrome and is characterized by prominent
cognitive dysfunction, behavioral and personality changes, and language
deficits. Patients with FTD progress rapidly and inexorably to death in
less than 10 years from its onset.

About AL001

AL001 is Alector's wholly owned human monoclonal antibody designed to
increase the levels of progranulin in the brain, a secreted immune
modulatory factor which when mutated leads to frontotemporal dementia
(FTD).

About Alector

Alector is a privately held biotechnology company whose mission is to
develop immuno-neurology and immuno-oncology therapies to treat
neurodegenerative diseases and cancer. Alector is headquartered in South
San Francisco, Calif. For more information, please visit www.alector.com.

View Comments and Join the Discussion!