Articles related to DEVICE/DRUG APPROVALS

RhoVac AB’s RV001 (onilcamotide) was granted Fast Track designation by FDA based on positive performance in its phase I/II trial. Unfortunately, in the follow-on Phase IIb BRaVac trial, the drug failed to demonstrate superiority over the placebo.

In the study,155 FDA-approved drugs with 136 potentially targetable genes were identified.One-hundred forty-six (94%) of the drugs showed no or low genetically predicted drug response. Although ATC-carrying BRAF mutations can benefit from BRAF inhibitors and this effect might be enhanced by a combined strategy including PIK3CA inhibitors in some of the patients, alterations in BRAFWT ATC are not directly targeted by currently FDA-approved options.

Hutchmed’s submission was based primarily on two phase 3 trials conducted in China. Even though a bridging study conducted in the US that suggested safety and efficacy similar to that observed in the Chinese study population, FDA responded that a multiregional clinical trial “more representative of the US patient population and in accordance with US medical practice will be required” for resubmission.

Mounjaro (tirzepatide) is a first-in-class, once-weekly, dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist that integrates the actions of both incretins into a single molecule. It enhances first- and second-phase insulin secretion, and reduces glucagon levels, both in a glucose-dependent manner. The FDA approval of Mounjaro as an adjunct therapy to diet and exercise for adults with type 2 diabetes was based on clinical trials where treatment with Mounjaro resulted in statistically significant reduction in both A1c and weight vs. semaglutide, insulin degludec, and insulin glargine.

Lilly Pharmaceutical’s Mounjaro (tirzepatide) is a once-weekly injection that promotes weight loss by mimicking the effects of incretins. This combination of a GLP-1 and a glucose-dependent insulinotropic polypeptide (GIP) yields weigh loss results comparable to surgical options.

A newly developed prolyl hydroxylase inhibitor (agents that increase endogenous erythropoietin production) holds the promise of improving outcomes for patients with anemia of chronic kidney disease. Randomized controlled trials have found these drugs to be at least as effective as erythropoiesis-stimulating agents.