New FDA Specialty Approvals

Oct. 7: The FDA expanded the label of BioMarin Pharmaceutical Inc.’s Palynziq (pegvaliase-pqpz) to increase the maximum allowable dose to 60 mg for the treatment of adults with phenylketonuria. The agency initially approved the drug with a maximum dose of 40 mg on May 24, 2018 (RSP 6/18, p. 8). The recommended initial dosing of the phenylalanine (Phe)-metabolizing enzyme is 2.5 mg subcutaneously once weekly for four weeks. Then dosage should be titrated in a stepwise manner over at least five weeks to achieve a dosage of 20 mg once daily. That can be increased to 40 mg once daily after 24 weeks in people who have not achieved blood Phe control. Dosing can be increased to 60 mg once daily in people who have been on the 40 mg dose for at least 16 weeks and not achieved blood Phe control. Website Drugs.com lists the price of one syringe for all three doses — 2.5 mg/0.5 mL, 10 mg/0.5 mL and 20 mg/mL — as $541.94. Visit www.palynziq.com.

Oct. 14: The FDA gave another approval to Harmony Biosciences Holdings, Inc.’s Wakix (pitolisant) for the treatment of cataplexy in adults with narcolepsy. The agency initially approved the selective histamine 3 (H3) receptor antagonist/inverse agonist on Aug. 15, 2019 (RSP 9/19, p. 8). Dosing in the first week is initiated with 8.9 mg once daily, then increased to 17.8 mg once daily in the second week and then increased to the maximum recommended dosage of 35.6 mg once daily in the third week. Website GoodRx lists the price of 60 17.8 mg tablets as more than $11,223. Visit https://wakix.com.

AIS Health Staff

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