MPR - Medical Professionals Reference

Jazz Pharmaceuticals announces US FDA approval of Ziihera ®

RegeneCyte is an allogeneic human umbilical cord blood-derived hematopoietic stem cell therapy. These hematopoietic progenitor cells divide and mature from the bone marrow and get released into the bloodstream where they restore blood counts and function.

Zevra Therapeutics announces US commercial availability of Miplyffa™ (arimoclomol) for treatment of Niemann-Pick disease type C. News release. Zevra Therapeutics. November 21, 2024.

Shorla Oncology announces FDA approval of Imkeldi (imatinib) oral solution, an oral liquid for the treatment of certain forms of leukemia and other cancers.

Common thyroid medicine linked to bone loss. News release. Radiological Society of North America. November 25, 2024.

Acoramidis is an oral small molecule designed to stabilize tetrameric transthyretin (TTR). The prescribing information indicates that among patients with ATTR-CM treated with the recommended dosage, near-complete in vitro TTR stabilization was observed as early as day 28 and through completion of the 30-month ATTRibute-CM study (ClinicalTrials.gov Identifier:

STS101 is an investigational DHE nasal powder product administered through a proprietary nasal delivery device. The formulation allows for fast absorption and quick self-administration to achieve high DHE plasma concentrations.

According to Soleno Therapeutics, the FDA required more time to review responses to recent information requests; no safety, efficacy or manufacturing concerns were raised.

Landiolol is a selective beta-1 adrenergic receptor blocker with a very short duration of action (elimination half-life is approximately 4 minutes). The approval was based on data from 5 randomized, double-blind, placebo-controlled trials that included 317 patients with

AXS-12 is an investigational highly selective potent norepinephrine reuptake inhibitor and cortical dopamine modulator. By modulating noradrenergic activity, AXS-12 is expected to promote wakefulness and improve cognition.