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FDA Approves New Combo Tx for Pompe – Pombiliti

………..catching up on recent FDA approvals

The FDA recently approved a new infused therapy, Pombiliti (cipaglucosidase alfa-atga) from Amicus Therapeutics, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy. 

Pombiliti is approved only in combination with Opfolda (miglustat), an oral drug that is to be administered approximately 1 hour before the infusion of Pombiliti. 

Pombiliti is a recombinant human GAA enzyme (rhGAA). Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. Pombiliti / Opfolda will be distributed in tandem. 

About 5,000 to 10,000 people have been diagnosed globally with Pompe. The majority of patients on current standard of care decline after about two years. The previous standard of care has been Sanofi’s Lumizyme. 

The combo therapy launched at an annual price of $650,000 for a patient weighing about 70 kg.

Pombiliti will be distributed by Orsini Specialty Pharmacy according to a recent press release.

Pombiliti was approved with a black box warning.

CLICK HERE to access prescribing information


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