From rxpgnews.com
FDA Approves Sodium Phenylacetate/Benzoate for Urea Cycle Disorder
By Akanksha, Pharmacology Correspondent
Feb 23, 2005, 11:17
Medicis has announced that U.S. Food and Drug Administration ("FDA") has approved AMMONUL(R) as an adjunctive therapy for the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle.
AMMONUL(R) is the only prescription drug available with the proven combination of 10% sodium phenylacetate and 10% sodium benzoate.
FDA granted it an orphan drug status with seven years of exclusivity based on long-term compassionate patient use in patients with Urea Cycle Disorder ("UCD").
UCD is an inherited, inborn error of metabolism. Patients with UCD lack one of the key enzymes that comprise the urea cycle, which can result in the life-threatening condition of hyperammonemia, i.e., dangerously heightened levels of ammonia in the bloodstream.
The drug is a hospital product administered intravenously as a rescue medication when a UCD patient progresses into a hyperammonemic crisis. It works by scavenging ammonia directly from the bloodstream, independent of the defective urea cycle.
BUPHENYL(R) (sodium phenylbutyrate), another Medicis prescription product indicated for UCD, is the only FDA-approved drug for maintenance treatment of UCD patients in the U.S., sodium phenylbutyrate tablets and powder are used for daily, life-long maintenance in UCD patients and work much the same as sodium phenylacetate and sodium benzoate, by scavenging ammonia from the bloodstream.
The annual incidence rate of UCD in the United States is approximately one in 30,000 live births. Historically, less than 5% of UCD patients survived the disorder, as the infant patient would appear simply septic. With the increasing awareness of UCD and the availability of this new drug as a rescue medication, statistics have improved drastically to over a 90% survival rate.
"We are pleased to announce FDA's approval of sodium phenylacetate and sodium benzoate therapy, the only acute care medication for Urea Cycle Disorder available in the U.S.," said Jonah Shacknai, Chairman and Chief Executive Officer of Medicis. "FDA's acknowledgment of the safety and efficacy of this important rescue drug is great news for patients, and will prove significant for Medicis. This new drug joins sodium phenylbutyrate as the only other drug approved to treat this rare and life-threatening disorder, making Medicis the only company offering a complete UCD treatment therapy."
The said drug is available by prescription and administered intravenously in the hospital setting. Although rare, vomiting, hyperglycemia, hypokalemia, convulsions and mental impairment have been reported with sodium phenylacetate and sodium benzoate therapy.
The drug is contraindicated for use by patients with known hypersensitivity to sodium phenylacetate and sodium benzoate. When used as directed, it is safe and efficacious as adjunctive therapy for the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the area cycle. Full prescribing information for the can be obtained by contacting Medicis.
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