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Crinecerfont

New Drug Approvals

2021 Nov 30;186(1):R1-R14. gmol 1 3D model ( JSmol ) Interactive image show SMILES show InChI Prete A, Auchus RJ, Ross RJ: Clinical advances in the pharmacotherapy of congenital adrenal hyperplasia. Eur J Endocrinol. doi: 10.1530/EJE-21-0794.

FDA 62
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Landiolol

New Drug Approvals

Pharmacokinetics of landiolol hydrochloride, a new ultra-short-acting beta-blocker, in patients with cardiac arrhythmias” Clinical Pharmacology and Therapeutics. 28 October 2021. February 2021). 32 (2): 828. doi : 10.1016/j.joa.2015.09.002. 2015.09.002. PMC 4823575. PMID 27092187. August 2000). 68 (2): 14350.

FDA 62
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Vertex Bags Experimental Drug for Rare Liver Disease after Disappointing Phase 2 Results | 2020-10-15

The Pharma Data

The study, which involves approximately 50 patients with the rare liver and lung disease, raised concerns about the experimental treatment’s safety and pharmacokinetic profile. That trial is continuing to enroll and dose patients, and results are expected in the first half of 2021. James Miessler.

Disease 52
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Everest Medicines Announces Amended Agreement with Spero Therapeutics

The Pharma Data

Spero plans to initiate additional Phase 1 studies to assess the penetration of SPR206 into the pulmonary compartment and pharmacokinetics in subjects with renal impairment in 2021. Spero will assign relevant SPR206 patents to Everest Medicines in Greater China, South Korea and certain Southeast Asian countries. SHANGHAI , Jan.

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Eplontersen

New Drug Approvals

“Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis” British Journal of Clinical Pharmacology. World Health Organization (2021). PMC 10540057. PMID 37768671. Diep, John K.; Yu, Rosie Z.; Viney, Nicholas J.; December 2022).

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Inozyme Pharma Announces Authorization to Proceed in U.S. and U.K. with Phase 1/2 Clinical Trial of INZ-701 for the Treatment of ENPP1 Deficiency

The Pharma Data

04, 2021 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. The Company expects to enroll the first subject in the first half of 2021 and provide preliminary safety and biomarker data in the second half of 2021. and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University.

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Roche provides update on tominersen programme in manifest Huntington’s disease

The Pharma Data

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). In December 2017, Roche licensed the investigational molecule from Ionis Pharmaceuticals.

Disease 52