Vimizim approved for rare childhood disorder

(HealthDay)—Vimizim (elosulfase alfa) has been approved by the U.S. Food and Drug Administration to treat a rare childhood disorder called Mucopolysaccharidosis Type IVA, also known as Morquio A syndrome.

The disorder is caused by a missing that leads to problems with , growth and movement, the agency said in a news release. It affects about 800 people in the United States.

Vimizim replaces the missing enzyme, known as GALNS. The drug's safety and effectiveness were established in clinical trials involving 176 people, ranging in age from 5 to 57. The most common side effects included fever, vomiting, headache, nausea, , chills and fatigue, the FDA said.

The drug's safety and effectiveness weren't evaluated in children under age 5 years, the agency added. Vimizim's label will include a boxed warning to include the risk of anaphylaxis, an allergic-like reaction that could be life threatening.

Vimizim was the first drug granted a Rare Pediatric Disease Priority Review Voucher, an FDA effort to encourage development of new treatments for rare childhood diseases.

The drug is marketed by BioMarin Pharmaceuticals, based in Novoto, Calif.

More information: The FDA has more about this approval.

add to favorites email to friend print save as pdf

Related Stories

Iclusig approved for rare leukemias

Dec 16, 2012

(HealthDay)—Iclusig (ponatinib) has been approved by the U.S. Food and Drug Administration to treat two rare forms of leukemia..

Opsumit approved for pulmonary arterial hypertension

Oct 23, 2013

(HealthDay)—Opsumit (macitentan) has been approved by the U.S. Food and Drug Administration to treat adults with pulmonary arterial hypertension, a debilitating disease characterized by high blood pressure in the lung arteries.

Xgeva approved for rare, non-malignant tumor

Jun 13, 2013

(HealthDay)—Xgeva (denosumab) has been approved by the U.S. Food and Drug Administration to treat giant cell tumor of the bone (GCTB), a rare tumor that's most often non-cancerous.

Imbruvica approved for mantle cell lymphoma

Nov 13, 2013

(HealthDay)—Imbruvica (ibrutinib) has been approved by the U.S. Food and Drug Administration to treat mantle cell lymphoma (MCL), a rare but aggressive form of blood cancer.

Recommended for you

Determine patient preferences by means of conjoint analysis

12 hours ago

The Conjoint Analysis (CA) method is in principle suitable to find out which preferences patients have regarding treatment goals. However, to widely use it in health economic evaluations, some (primarily methodological) issues ...

FDA approves hard-to-abuse narcotic painkiller

Jul 25, 2014

(HealthDay)—A new formulation of a powerful narcotic painkiller that discourages potential abusers from snorting or injecting the drug has been approved by the U.S. Food and Drug Administration.

Race affects opioid selection for cancer pain

Jul 25, 2014

(HealthDay)—Racial disparities exist in the type of opioid prescribed for cancer pain, according to a study published online July 21 in the Journal of Clinical Oncology.

FDA approves tough-to-abuse formulation of oxycodone

Jul 25, 2014

(HealthDay)—Targiniq ER (oxycodone hydrochloride and naloxone hydrochloride extended release) has been approved by the U.S. Food and Drug Administration as a long-term, around-the-clock treatment for severe ...

Tough-to-abuse formulation of oxycodone approved

Jul 25, 2014

(HealthDay)—Targiniq ER (oxycodone hydrochloride and naloxone hydrochloride extended release) has been approved by the U.S. Food and Drug Administration as a long-term, around-the-clock treatment for severe pain when other ...

User comments