A Prescription Drug User Fee Act target date of February 28, 2026 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics ...

A special diet is the main treatment for phenylketonuria. Medications are also available. Treatment and monitoring is lifelong. Phenylketonuria (PKU) is an inherited condition where the amino acid ...

- First marketing authorization submission for sepiapterin with additional global submissions to follow in 2024 - SOUTH PLAINFIELD, N.J., March 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: ...

NEW YORK (Reuters Health) - For women with the rare genetic disorder phenylketonuria, a new study highlights the importance of sticking with therapy before and during pregnancy. People with ...

On Friday, the U.S. Food and Drug Administration (FDA) approved BioMarin Pharmaceutical Inc.’s supplemental application for Palynziq (pegvaliase-pqpz), a treatment for phenylketonuria (PKU) in ...

-- The lack of therapeutic alternatives creates a clear opportunity for new entrants to develop novel or complementary treatment options for PKU. -- Several leading companies, including Jnana ...

WARREN, N.J., July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the ...

Otsuka Pharmaceutical Development & Commercialization, Inc. and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced new ...

With several potential therapies, including Sepiapterin (PTC Therapeutics (PTCT)), NGGT002 (NGGT), JNT-517 (Jnana Therapeutics), Pegvaliase (BioMarin), and others under investigation, the ...