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Selected Product Category: Rare Diseases and Specialized Treatments

Myalept (metreleptin) � SC injection, daily, for complication of leptin deficiency in generalized lipodystrophy


Myalept

Recombinant leptin analog

Luxturna (voretigene neparvovec) � subretinal injection (one-time per eye)


Luxturna

Gene therapy (AAV2)

Folotyn (pralatrexate) � IV push weekly (cycles), for relapsed or refractory peripheral T-cell lymphoma


Folotyn

Antifolate chemotherapy

Brineura (cerliponase alfa) � intraventricular infusion every 2 weeks


Brineura

Enzyme replacement therapy

Blincyto (blinatumomab) � for B-cell precursor acute lymphoblastic leukemia (ALL) including MRD-positive disease


Blincyto

Bispecific T-cell engager

Ravicti (glycerol phenylbutyrate) � oral liquid, multiple doses per day, for chronic management of urea cycle disorders


Ravicti

Nitrogen-scavenging agent

Zokinvy (lonafarnib) � oral capsules, twice daily with food


Zokinvy

Farnesyltransferase inhibitor

Danyelza (naxitamab-gqgk) � IV infusion cycles with GM-CSF support


Danyelza

Anti-GD2 monoclonal antibody

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