The Food and Drug Administration on Tuesday approved a new capsule-based drug to treat Gaucher's disease.
Regulators
approved the new drug, Cerdelga, for patients with the Type 1 form of
the rare genetic disorder, which causes excess fat to build up in the
spleen, liver and bone marrow.
Cerdelga is from
Genzyme, the specialty drugmaker that introduced the first drug for
Gaucher's disease 20 years ago. The oral treatment could serve as an
alternative to Genzyme's best-selling drug Cerezyme, which is given
intravenously.
Cerezyme had sales of $935 million
last year. The drug costs $300,000 for a year's supply, on average.
Genzyme says it will soon release pricing for its new drug.
Type 1 is the most common form of Gaucher's disease and affects an estimated 6,000 people in the U.S.
Patients
with Gaucher's disease lack a certain enzyme that helps the body break
down fat. Cerdelga works by reducing levels of the fat that collect in
tissue and organs. It differs from Cerezyme, which is a man-made
replacement for the enzyme that patients need to break down fat in the
body. Cerezyme is administered by drug infusion every two weeks. The
drug was introduced in 1994 and launched Genzyme into the market for
rare disease drugs.
Genzyme, which is based in
Cambridge, Massachusetts, is a subsidiary of French drugmaker Sanofi,
which acquired the company in 2011.
تعليقات
إرسال تعليق