#fda-approval-criteria

#fda-approval-criteria

We've created a small molecule chemical which gets in your body very well. It can mimic the effects of the peptide and can be taken more conveniently any time of day without any food or water restrictions.

A report last year found unnecessary surgeries were carried out, cancers were missed and poor standards of care were delivered at the University Hospital of North Durham and Darlington Memorial Hospital. CDDTF said it wanted to support the patients it had let down, including by offering access to psychological support, and to ensure they knew how to make a claim or raise concerns with police.

RBC Capital analyst Luca Issi upgraded the stock to Outperform from Sector Perform with a price target of $35, up from $11. Wells Fargo also upgraded uniQure to Overweight from Equal Weight with a $60 price target. The catalyst: the departure of Vinay Prasad from the FDA. RBC views this as a positive for uniQure, noting it is "not inconceivable" that the FDA reverts to its prior stance, and believes Prasad's departure is likely to open up a more balanced discussion on risk/reward for Huntington's disease.

According to the patent, a specific crystalline form of the drug known as polymorph C may be more effective than other versions because it is absorbed more efficiently by the body. The patent also notes that laboratory studies showed the drug reduced tumor growth and helped mice with brain tumors live longer, prompting early clinical trials to test whether the treatment is safe and effective in humans.

Emma Dyer remembers the moment she clicked "buy now" on a set of weightloss jabs she found online. She had no medical consultation, no ID checks, and no questions about her history of anorexia and bulimia. "It was just so easy - too easy," she says. "They never asked for my medical history or what medication I was taking. It was like buying groceries."

The U.S. Food and Drug Administration is reportedly mulling whether more prescription drugs should be sold over the counter (OTC) at pharmacies. In an interview on Wednesday, FDA commissioner Martin Makary told CNBC that everything should be over the counter except drugs that are deemed unsafe or addictive or that require clinical monitoring. Makary said the agency is reviewing how it decides which drugs can be sold with or without a prescription from a health care practitioner.

After having "constructive conversations with stakeholders," Hims decided to stop offering the treatment, the company said in a post on X Saturday.

The news is the latest sign of the FDA's heightened scrutiny of vaccines under Health Secretary Robert F. Kennedy Jr., particularly those using mRNA technology, which he has criticized before and after becoming the nation's top health official. Moderna received what's called a "refusal-to-file" letter from the FDA that objected to how it conducted a 40,000-person clinical trial comparing its new vaccine to one of the standard flu shots used today.

Prasad's tenure was generally marked by controversy, but he is departing amid a cluster of self-destructive decisions. Those include a shocking rejection of an mRNA vaccine (which was over the objections of agency scientists and quickly reversed); a demand for an additional clinical trial on a gene therapy for Huntington's disease, which was widely seen as moving the goalpost for the therapy; his startling choice to publicly attack the maker of that gene therapy, UniQure; and alleged abuse of FDA staff, who say he created a toxic work environment.

This week in Other Barks & Bites, IPWatchdog's IP news roundup: the House of Representatives passes drug patent legislation, while antitrust legislation targeting patent-related activities is introduced into the Senate and the Trump administration mandates pricing information for pharmaceutical ads; the Patent Trial and Appeal Board (PTAB) issues a pair of precedential decisions on cases with multiple petitions; the USPTO issues marijuana-related trademark guidelines and a notice on modifying patent term adjustment practices;

While the move came as a surprise to the high-profile vaccine maker, it is just the latest hostility toward vaccines-and mRNA vaccines in particular-from an agency overseen by the fervent anti-vaccine activist Robert F. Kennedy Jr. In his first year in office, Kennedy has already dramatically slashed childhood vaccine recommendations and canceled $500 million in research funding for mRNA vaccines against potential pandemic threats.

Public health consultant Dr Ross Keat said supporting people earlier to make small preventative changes would make "a big difference later on". Some 3,500 people in the north of the island within that age bracket are eligible for the checks. The checks will be carried out by two pre-existing nurses that support GP staff and would not replace GP appointments, Keat explained, adding that the cost would be minimal and absorbed by Ramsey Group Practice.

EMPAVELI is the first and only approved treatment for C3G and IC-MPGN across pediatric patients 12+, adults, and post-transplant recurrence. That's roughly 5,000 patients in the U.S., with EMPAVELI holding exclusive approval for about two-thirds. Add the European CHMP positive opinion in December 2025, and you have a rare disease franchise with global expansion potential and pricing power that typically commands gross margins north of 90%.

We provide thought partnership. When a company is developing a drug, there's a lot of work involved, such as understanding the science, designing a study and generating good data. We come in and explain what the standard of care looks like today for their patient population, and what we think it will look like in five to eight years or whenever they plan to launch their therapy.

The cancer was fastacting, and if I'd left it even six months, the outcome could have been much worse,

Telehealth company Hims & Hers dropped its plan to offer a knockoff version of the weight-loss pill Wegovy on Saturday - two days after it announced the new drug and one day after the Food and Drug Administration threatened to restrict access to the ingredients needed to copy popular weight-loss medications. Hims had said Thursday that it would offer a compounded version of the new Wegovy pill that drugmaker Novo Nordisk just began selling last month.

Many researchers are surprised and relieved over an unusual step taken by the US National Institutes of Health (NIH): the agency is rolling back the red tape on a host of basic-science experiments that involved human participants and had been classified as clinical trials. The decision, which was announced on 29 January and is part of a broader NIH effort to reduce administrative burden, should free such research from the heavy bureaucratic requirements that are designed for clinical trials but are sometimes ill-suited to other fields, such as basic psychology and behavioural studies.