Hyperphenylalaninemia

Vertex Pharmaceuticals, Amgen and Jazz Pharmaceuticals are among companies that received a positive opinion from European regulators for drugs that are already in the US market. The European Medicines Agency’s human medicines committee (CHMP) said on Friday that it recommended the European Commission to give the go-ahead for six new medicines and 10 label expansions. Among the six new medicines was Vertex’s cystic fibrosis oral therapy called Alyftrek, which was approved by the FDA last December for the same indication. Amgen similarly got a CHMP nod for Tepezza in moderate to severe Thyroid Eye Disease. Tepezza was approved by the FDA for that condition in January 2020. Jazz also secured a positive opinion for its bile duct cancer antibody Ziihera, which was approved by the FDA in November last year. On Friday, the committee said it would re-examine its earlier decision to reject Eli Lilly’s Alzheimer’s drug Kisunla. It also recommends PTC Therapeutics’ drug, dubbed Sephience, to be used to treat hyperphenylalaninemia in patients with the metabolic disease phenylketonuria. The other CHMP medicine recommendations are for Italfarmaco’s therapy Duvyzat for Duchenne muscular dystrophy and Purpose Pharma’s Attrogy for ATTR amyloidosis. As for the 10 label expansions, the committee recommended extending two of Bayer’s drugs, including a new dose of Adempas, which is approved for pulmonary arterial hypertension, as well as extending the label for the hemophilia A drug called Jivi for children aged seven and above. Other label recommendations: The regulators also recommended the EC approve nine new biosimilar products, eight of which were biosimilars to Amgen’s denosumab, which is a treatment for rare bone disease.

Otsuka Pharmaceutical Co., Ltd. (Otsuka) announces that it has completed the acquisition of Jnana Therapeutics Inc. (Jnana), based in Boston, Massachusetts. Jnana has become a direct subsidiary of Otsuka America, Inc. (OAI), which is a fully owned subsidiary of Otsuka.  Based on the terms of the agreement signed in August of this year, Otsuka paid (U.S.) $800 million to the shareholders of Jnana as consideration for the acquisition of all outstanding shares of the company. In addition, Otsuka may pay up to (U.S.) $325 million to Jnana shareholders in development and regulatory milestone payments based on the progress of future development products. Jnana utilizes an innovative drug discovery approach, the Reactive Affinity Probe Interaction Discovery (RAPID) platform, for a variety of drug targets on which it has been difficult to obtain small-molecule drugs through traditional screening systems. With its focus on identifying first-in-class compounds for autoimmune and rare diseases, Jnana is in a unique competitive position. In the autoimmune disease field, the company is pursuing small molecule drug discovery for highly validated targets such as interferon regulatory factor 3 (IRF3), a master transcription factor for the production of interferon. In addition, using this drug discovery technology, Jnana developed JNT-517, an oral small-molecule inhibitor of a protein that regulates phenylalanine reabsorption in the kidneys, and is conducting a Phase 1b/2 study in patients with phenylketonuria (PKU), a rare, inherited metabolic disorder in which symptoms usually begin to appear in infancy. Earlier this month, Jnana presented the results of the Phase 1b/2 study at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium. In addition to data for the 75-mg dose, new data for the 150-mg dose (both twice daily) were disclosed at this conference presentation.  JNT-517 has received orphan drug designation and rare pediatric disease designation from the U.S. FDA and orphan designation from the European Medicines Agency (EMA) for the treatment of PKU. JNT-517 has also been granted eligibility for the EMA PRIME (PRIority MEdicines) scheme for the treatment of hyperphenylalaninemia. Based on the data from this Phase 1b/2 study, the company is currently engaging in discussions with U.S. and European regulatory authorities and plans to initiate a Phase 3 study in early 2025.