Ultragenyx Pharmaceutical Gets FDA Approval for Mepsevii

01:09 11/15/2017

Shares of Ultragenyx Pharmaceutical Inc. RARE  are yet to significantly react to the news of FDA approval for Mepsevii used to treat pediatric and adult patients with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs.

The FDA granted Mepsevii a Fast Track designation, which seeks to accelerate the development and review of drugs that are intended to treat serious conditions where initial evidence showed the potential to address an unmet medical need.

Mepsevii also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Fewer than 150 patients worldwide are affected by mucopolysaccharidosis. The safety and efficacy of Mepsevii were established in clinical trial enrolling a total of 23 patients ranging from 5 months to 25 years of age. Patients received treatment once every two weeks for up to 164 weeks.

Common side effects after treatment with Mepsevii include infusion site reactions, diarrhea, rash and anaphylaxis.

The features of MPS VII vary widely from patient to patient, but most patients have various skeletal abnormalities that become more pronounced with age, including short stature.

Affected individuals can also develop heart valve abnormalities, enlarged liver and spleen, and narrowed airways which can lead to lung infections and trouble breathing. The life expectancy of individuals with MPS VII depends on the severity of symptoms. Some affected individuals do not survive infancy, while others may live into adolescence or adulthood.

Heart disease and airway obstruction are major causes of death in people with MPS VII. Affected individuals may have developmental delay and progressive intellectual disability.

This article has been provided by a Chasing Markets contributor. All content submitted by this author represent their personal opinions, and should be considered as such for entertainment purpose only. All opinions expressed are those of the writer, and may not necessarily represent fact, opinions, or bias of Chasing Markets.
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