FDA+ channel feed

FDA warns anes­the­si­ol­o­gist for run­ning clin­i­cal in­ves­ti­ga­tions with­out an IND

Maggie Jeffries, owner of Houston-based Avanti Anesthesia, has run afoul of the FDA’s rules on clinical investigations, according to a warning letter released today, with the agency noting that the combo of drugs she used put some surgery patients at increased risks.

The letter pertains to two clinical investigations: one of which Jeffries served as sponsor-investigator for a trial with cataract surgery patients, and the other she performed for the drug compounder Imprimis Pharmaceuticals.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck asks EMA to reeval­u­ate re­ject­ed Covid-19 an­tivi­ral

Merck is seeking a reevaluation of the European Medicines Agency’s (EMA) rejection of the pharma’s blockbuster Covid-19 pill, known as Lagevrio (molnupiravir), which has served as a second option to Pfizer’s Paxlovid (nirmatrelvir/ritonavir).

The EMA announced its long-awaited rejection in late February, saying publicly:

Based on the totality of data, it was not possible to conclude that Lagevrio can reduce the risk of hospitalisation or death or shorten the duration of illness or time to recovery in adults at risk of severe disease. Furthermore, it was not possible to identify a specific group of patients in whom a clinically relevant benefit of Lagevrio could be demonstrated.

FDA paus­es Mer­sana’s clin­i­cal tri­al af­ter pa­tient dies

The FDA has paused Mersana Therapeutics’ Phase I clinical trial for a cancer treatment after a patient died, the biotech said Monday morning.

The death was deemed to be related to its drug, the Cambridge, MA-based biotech said.

The patient who died was the second to be dosed in Mersana’s clinical trial for a STING agonist — a drug target that scientists believe can boost immune response against tumors but has seen a number of challenges over the years, from low efficacy to nixed programs.

Renee Aguiar-Lucander, Calliditas CEO

Cal­lid­i­tas plans to file for full ap­proval for rare kid­ney dis­ease drug fol­low­ing PhI­II da­ta

Calliditas believes Phase III confirmatory data it released Sunday for its oral steroid to treat a rare kidney disease could be the final piece needed to get full approval from the FDA.

The trial studied Nefecon, the biotech’s delayed-release budesonide capsules marketed as Tarpeyo, on primary IgA nephropathy (IgAN) patients who were also on a RAS inhibitor therapy. Tarpeyo scored accelerated approval from the agency in December 2021 and conditional marketing authorization from the European Commission in July 2022.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.

Steve Davis, Acadia Pharmaceuticals CEO

Up­dat­ed: Aca­dia wins first FDA ap­proval for Rett syn­drome drug

The FDA on Friday approved Acadia Pharmaceuticals’ treatment for Rett syndrome, a rare neurodevelopmental disorder, which will be sold under the brand name Daybue (trofinetide), the company announced.

The average list price for the drug will be about $575,000 to $595,000 a year, CEO Steve Davis said on a conference call Monday morning. That comes from a $21.10 per milliliter price that’s used for weight-based dosing. The company’s “net realized cost” will be approximately $375,000, he said.

Re­pub­li­can sen­a­tors slam pro­posed ac­cel­er­at­ed ap­proval pay­ment re­forms

Republican senators are speaking out against a proposed test model in which drugmakers would effectively make less for drugs approved via the FDA’s accelerated approval.

The test program, dubbed the Accelerating Clinical Evidence Model, was announced last month as a way to incentivize quicker confirmatory trials and “reduce Medicare spending on drugs that have no confirmed clinical benefit.” HHS Secretary Xavier Becerra directed CMS to begin consultation with the FDA on the model this year.

FDA will phase out 22 Covid-19 guid­ance doc­u­ments as emer­gency de­c­la­ra­tion ex­pires

With the public health emergency and the national emergency declaration expected to expire on May 11, the FDA will be letting some guidance documents expire along with it.

In a document published Friday in the Federal Register, the FDA said that of the 72 Covid-19 guidances that are still active, 22 will no longer be in effect when the declarations expire.

Those guidance docs that are being cut, and which typically offered some leniency from the FDA due to the constraints of the pandemic, include ones related to manufacturing considerations for cell and gene therapies, nonhuman primate supply constraints, statistical considerations for clinical trials and more.

FDA sched­ules ad­comms for En­ta­sis' an­timi­cro­bial treat­ment, In­ter­cep­t's NASH hope­ful

The FDA this morning announced two upcoming advisory committee meetings in April and May — with the goal of reviewing two drugs with a lot of history that have gone through multiple setbacks.

According to notices published in the Federal Register, the FDA is convening its Antimicrobial Drugs Advisory Committee on April 17 to discuss Entasis Therapeutics’ NDA for sulbactam-durlobactam for the treatment of Acinetobacter infections.

GAO to FDA: Do more to en­cour­age drug man­u­fac­tur­ing in­no­va­tion

The Government Accountability Office on Friday released a report calling on the FDA to better encourage the adoption of advanced drug manufacturing practices, particularly as interviews with 15 stakeholders from industry said delays in adopting advanced tech are related to regulatory uncertainties.

In addition to not encouraging more advanced manufacturing practices, like continuous manufacturing, GAO found that the FDA “lacks information on the extent to which its industry engagement and policy and guidance efforts encourage adoption of advanced manufacturing.” What’s more, the FDA has not even defined or documented its goals in this area, GAO found.

Albert Bourla, Pfizer CEO (Matthew Busch/Bloomberg via Getty Images)

Pfiz­er's mi­graine nasal spray from Bio­haven notch­es ap­proval

Pfizer’s nabbed its first approval out of its Biohaven acquisition with a nod for migraine spray Zavzpret on Friday. The FDA approval also marks a first intranasal delivery method among the newer class of calcitonin gene-related peptide (CGRP) receptor antagonists in migraine treatments.

Pfizer picked up zavegepant in its $11.6 billion acquisition of Biohaven, which also included Nurtec ODT and a preclinical portfolio of CGRP assets, finalized in October. Biohaven, or New Biohaven, was spun out as a separate company now developing the remaining non-CGRP assets, keeping the $BHVN ticker and continuing to be led by CEO Vlad Coric.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.