AMA Morning Rounds: FDA approves Genzyme's Lumizyme to treat patients with Pompe disease

The AP (5/26) reports that the Food and Drug Administration "approved Genzyme's drug to treat a muscle development disease." The drug, called Lumizyme (alglucosidase alfa), has just been approved to treat patients with Pompe disease in the United States and is currently manufactured in Belgium, where it is known as Myozyme.

The Boston Globe (5/26, Weisman) reports, "The decision represents the most important US drug approval for the Cambridge biotechnology company since 2003, when regulators signed off on Fabrazyme [agalsidase beta], a treatment for Fabry disease. Genzyme hopes Lumizyme will become a blockbuster, with worldwide sales of at least $1 billion annually."

The Boston Herald (5/26, McConville) reports, "The Cambridge biotechnology company said the drug will be sold in a box that warns users of potentially life-threatening anaphylactic reactions, severe allergic reactions and immune-mediated reactions." According to the company, it "will only give the drug to patients who are eight or older and enrolled in a risk mitigation program."

AMA Morning Rounds: Physicians using novel procedures for pain management.

Laura Landro writes in the Wall Street Journal (5/11) The Informed Patient column that peripheral-nerve stimulation, a procedure which allows a physician to implant a tiny, battery-operated generator, is one of a growing number of treatments for people who endure chronic pain, and who have been unable to find relief from drugs, physical therapy, or even acupuncture. The procedures typically cost between $15,000 and $50,000. Data show that about 76 million Americans suffer from chronic pain. Notably, the CDC has said that over eight million Americans use pain management drugs.

AMA Morning Rounds: FDA grants orphan drug status to potential ALS treatment.

The AP (3/10) reported, "Cytokinetics Inc. said Wednesday regulators have granted 'orphan drug' status to its potential" amyotrophic lateral sclerosis, or "Lou Gehrig's disease treatment." The company also "said it plans to start a mid-stage clinical trial in the first half of this year to test the drug's effectiveness." Reuters (3/10, Sengupta) noted that the drug is called CK-2017357.

AMA Morning Rounds: FDA approves Botox to treat elbow, wrist, and finger spasms.

The AP (3/10, Perrone) reports that "the Food and Drug Administration said Tuesday it approved Allergan's...Botox [Botulinum toxin Type A] to treat spasms of the elbow, wrist and fingers." The agency "stressed in a statement that Botox is not approved to treat spasms in larger muscles of the arms or legs." In 2009, "the agency added warnings to Botox about its potential to migrate from limbs to other parts of the body, causing breathing problems."

Bloomberg News (3/10, Peterson) reports that "clinical trials showed the drug helped patients with muscle spasms in the upper limbs, Russell Katz, director of the Food and Drug Administration's Division of Neurology Products, said...in an e-mailed statement." Dow Jones Newswire (3/10, Dooren), Reuters (3/10), and HealthDay (3/9, Roberts) also covered the story.

AMA Morning Rounds: Bristol-Myers Squibb, Allergan to jointly develop drug to treat neuropathic pain

The AP (3/3) reported that "Bristol-Myers Squibb and Allergan will jointly attempt to develop a drug to treat chronic pain from tissue damage, the drugmakers said." According to the AP, "the drug is said to be ready for mid-stage human trials as a treatment for neuropathic pain, or chronic pain usually accompanied by tissue injury. It is common in people with arthritis, cancer, and nerve damage."

The Orange County Business Journal (3/3) reported that "Bristol-Myers gains the rights to develop, make and market the compound," currently known as AGN-209323, "for just about all uses except for those related to the eye, which Allergan retains." Allergan will receive "an initial payment of $40 million and potential payments of up to $373 million if the compound is successful." Reuters (3/3) and the Wall Street Journal (3/3, Becker) also covered the story.