Specialty Pharmacy

NEW YORK — The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.

Pfizer and Protalix BioTherapeutics announced Wednesday the approval of Elelyso (taliglucerase alfa), an enzyme-replacement therapy for the long-term treatment of Type 1 Gaucher disease.

NEW YORK — An online plea for help from a mother dying of breast cancer prompted the manufacturer of an experimental drug to release it early, according to published reports.

In an article posted on its website, CNN told the story of 46-year-old Darlene Gant, a woman with stage-four breast cancer, as she wrote letters to be given to her 11-year-old son on his upcoming birthday, and at future events, such as his high school and college graduations and marriage.

WHAT IT MEANS AND WHY IT'S IMPORTANT — Since the development of the first smallpox vaccine in 1796, vaccines have saved countless lives around the world and helped to make such deadly scourges as smallpox and polio things of the past.

(THE NEWS: PhRMA report lists almost 300 vaccines under development. For the full story, click here.)

PHILADELPHIA — The Food and Drug Administration has approved a drug made by GlaxoSmithKline for certain cancer patients.

EAST HANOVER, N.J. — A drug made by Novartis has received approval from the Food and Drug Administration to treat benign kidney tumors related to tuberous sclerosis complex.

The FDA approved Afinitor (everolimus) tablets for treating the tumors, known as renal angiomyolipomas, in adults with TSC who don't require surgery. The drug already was approved to treat brain tumors known as subependymal giant cell astrocytoma, or SEGA, in patients with TSC.

PHOENIX — Patients with a rare, life-threatening genetic immune system disorder can receive treatment for sudden episodes more quickly and cost-effectively at home than if they go to the emergency room, according to a Walgreens Infusion Services study being presented Wednesday at the National Home Infusion Association Annual Conference and Exposition here.

WASHINGTON — Drug makers in the United States are developing nearly 300 vaccines for preventing and treating diseases, according to a new report by a drug industry trade group.

THOUSAND OAKS, Calif. — Biotech drug maker Amgen is buying a pharmaceutical company based in Turkey, the companies said Wednesday.

Amgen said it would acquire 95.6% of Istanbul-based Mustafa Nevzat for $700 million in an all-cash deal, saying the purchase would allow it to expand in Turkey and the surrounding region. The company, also known as MN, is a major supplier of injectable drugs in Turkey and, increasingly, an exporter of medicines.