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The U.S. Food and Drug Administration said on Wednesday that it has approved Jazz Pharmaceuticals' (JAZZ.O), opens new tab drug to treat diffuse midline glioma, a rare and aggressive tumor, in adults and children aged one year and older. The approval expands Jazz’s cancer drug portfolio beyond its existing treatments for certain blood and lung cancers. This is the first FDA-approved systemic therapy for diffuse midline glioma with a specific mutation that has progressed despite prior treatments, the agency said. The drug, Modeyso, is expected to become available in the U.S. in the coming weeks and is administered as a once-weekly oral capsule. Diffuse midline glioma (DMG) is a rare and aggressive brain tumor that primarily affects children and young adults. It develops in the brain’s and spinal cord’s midline structures, such as the brainstem, thalamus, and spinal cord. An estimated 3,940 people are living with this tumor in the United States, according to NIH data. The FDA decision was based on data from 50 patients in five clinical studies, showing that the drug helped shrink tumors in about 22% of cases. Among those who responded, the benefit lasted a median of just over 10 months. "We think it fits very well in terms of addressing...

The U.S. Food and Drug Administration on Tuesday approved Ajovy, an injection made by Israeli drugmaker Teva Pharma (TEVA.TA), opens new tab, to help prevent migraines in children aged six and older who weigh 45 kilograms or more. This is the first time a drug has been approved for preventing migraines in children. The injection is given once a month. The most common side effects are pain and redness where the shot is given. Serious side effects include itchiness, rash and drug hypersensitivity, but the overall safety was similar to what was seen in adult migraine studies, the FDA said. Episodic migraine refers to recurring severe headaches in children that do not occur daily but are frequent enough to interfere with daily activities. These headaches are often accompanied by symptoms such as nausea, fatigue and sensitivity to light and sound. Ajovy is part of a class of drugs called CGRP inhibitors, which block a protein involved in triggering migraines. The treatment was first approved for adults in 2018 and competes with similar drugs such as Amgen's (AMGN.O), opens new tab Aimovig and Eli Lilly's (LLY.N), opens new tab Emgality.

The FDA approves aceclidine ophthalmic solution 1.44%, the first aceclidine eye drop for presbyopia, offering a new solution for millions struggling with near vision loss. The FDA approved aceclidine ophthalmic solution 1.44% (Vizz; Lenz Therapeutics) for the treatment of presbyopia in adult patients. With this action, Vizz has become the first and only FDA-approved aceclidine-based eye drop for this condition, according to a news release. Vizz is an aceclidine-based eye drop that differentiates itself from others with its mechanism of action. It is a predominantly pupil-selective miotic that interacts with the patient’s iris, with minimal ciliary muscle stimulation. The eye drop contracts the iris’s sphincter muscle, resulting in a pinhole effect that uniquely achieves a sub-2mm pupil that extends a patient’s depth of focus to significantly improve their near vision without resulting in a myopic shift. Presbyopia is the inevitable loss of near vision associated with aging. It impacts the daily lives of nearly all people over the age of 45. The crystalline lens in individuals' eyes gradually hardens and becomes less able to change shape as they age, and this loss of elasticity of the lens reduces its ability to focus incoming light from near objects onto the retina. Adults over...

The U.S. Food and Drug Administration has extended its review of GSK's (GSK.L), opens new tab blood cancer drug, Blenrep, as a combination treatment, the company said on Wednesday. The health regulator has set an action date of October 23, which provides the FDA with time to review additional information provided in support of the application. Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here. The company is seeking approval for Blenrep in combination with other treatments for patients with relapsed or refractory multiple myeloma who had received at least one prior therapy. The regulator's panel of independent experts last week recommended against the drug, citing concerns about previously documented risks of eye-related side effects. The "odds of approval have clearly gone up (from a near zero after the advisory committee)," Barclays analyst Emily Field said, adding that investors were bracing for a negative decision from the FDA. U.S.-listed shares of the British drugmaker were up nearly 3% at $38.06. The experts voted 5-3 to say the benefits of Blenrep with bortezomib and dexamethasone at the proposed dosage did not outweigh its risks, and 7-1 against Blenrep in combination with pomalidomide and dexamethasone. Bortezomib and pomalidomide are...

The U.S. Food and Drug Administration's panel of independent advisers on Friday voted against the efficacy of Otsuka Pharma's (4578.T), opens new tab antipsychotic drug in combination with Viatris' (VTRS.O), opens new tab antidepressant Zoloft for treating adults with PTSD. The panel voted 10-1 to say the available data does not establish the efficacy of the drug, brexpiprazole, in combination with Zoloft, chemically known as sertraline, for treating the condition. Its decision is in line with the FDA staff reviewers' assessment on Wednesday. Otsuka's application was based on the data from one mid-stage and two late-stage studies testing the combination treatment compared to sertraline plus placebo. It had failed to meet the main goal of significantly reducing severity of PTSD symptoms in one of the late-stage studies. PTSD, a disorder caused by very stressful events, affects about 4% of U.S. adults and is commonly associated with war veterans. But natural disasters, abuse or other trauma may also trigger the condition in civilians. "We simply cannot negate one negative study and say we are going to approve based on two other positive studies," said panelist Pamela Shaw, biostatistician at Kaiser Permanente. The advisers discussed possible side effects such as weight gain and movement disorders associated...

The U.S. Food and Drug Administration's staff reviewers on Tuesday raised safety concerns that GSK's (GSK.L), opens new tab blood cancer drug Blenrep, when used in combination with other treatments, may cause eye damage in patients. In briefing documents released on the health regulator's website, staff reviewers said the benefit-risk profile of Blenrep remains unclear, citing concerns about safety, tolerability and appropriate dosages. The drug causes ocular toxicity that represents "unique toxicity not seen with any currently available treatments for multiple myeloma," staff reviewers said. The FDA staff's assessment comes ahead of a meeting of its independent experts on Thursday.

In a sweeping enforcement campaign aimed at protecting consumer health, the Saudi Food and Drug Authority (SFDA) has taken decisive action on two separate fronts — seizing 1.5 million non-compliant cosmetic products stored illegally in a residential facility and suspending a European pharmaceutical factory after uncovering serious violations of manufacturing standards. In Najran, SFDA inspectors shut down an unauthorised warehouse disguised as a residential facility, where approximately 1.5 million units of cosmetic products with manipulated expiration dates were being stored and distributed. The items, which violated national safety regulations, posed a direct risk to consumer health. The authority has referred the case to the Public Prosecution for further legal action. “This type of fraud represents a serious threat to public safety and will not be tolerated,” the SFDA said in a statement, adding that it remains committed to combating all forms of non-compliance in the cosmetics sector. Under Article 31 of the Kingdom’s Cosmetics Law, offences such as tampering with expiration dates or distributing falsified products can result in up to five years in prison, fines of up to SR5 million or both. In a separate case, the SFDA announced the suspension of a European pharmaceutical manufacturer’s registration following an external inspection that revealed...

The U.S. Food and Drug Administration approved a pre-filled syringe version of GSK's (GSK.L), opens new tab blockbuster shingles vaccine Shingrix, providing patients a more convenient option to receive the shot, the company said on Thursday. Shingles is a viral infection that causes a painful rash anywhere on the body and can sometimes lead to serious complications such as long-term nerve pain and vision loss. The infection is caused by the varicella-zoster virus (VZV), a type of herpes virus that remains in patients' bodies after they have had chickenpox and reactivates years later. The previous version of GSK's vaccine required a healthcare professional to mix the powdered form of the virus proteins with a liquid that boosts the immune response, before administration. The new form of Shingrix, which does not require the mixing, was approved based on data that showed both versions were equally effective.

The U.S. Food and Drug Administration's reviewers on Wednesday raised efficacy concerns over the use of Otsuka Pharma's (4578.T), opens new tab drug in combination with Viatris' (VTRS.O), opens new tab Zoloft for the treatment of adults with PTSD. The FDA staff's assessment comes ahead of a meeting of its independent experts on Friday, who will make recommendations on whether the regulator should approve the use of the drug, brexpiprazole, in combination with Zoloft for treating PTSD. The FDA staff cited inconsistent trial results and a modest treatment effect that may not be clinically meaningful as reasons for the efficacy concerns. Otsuka's application was based on the data from one mid-stage and two late-stage studies testing the combination of brexpiprazole and Zoloft in PTSD patients compared to sertraline monotherapy. The reviewers, however, noted that one of the late-stage studies failed to show statistically significant differences in treatment response between the two groups. They also added that the mid-stage study had statistical and methodological concerns. PTSD is a mental health condition that can develop after a traumatic event, causing symptoms including flashbacks, nightmares and severe anxiety. Brexpiprazole, sold under the brand name of Rexulti, is approved in the U.S. to treat agitation in patients with Alzheimer's disease...

KalVista Pharmaceuticals (KALV.O), opens new tab said on Monday the U.S. Food and Drug Administration has approved its drug for a type of hereditary swelling disorder, sending its shares up 33.3% in premarket trading. The regulator's greenlight comes after it extended the review of the drug, Ekterly, due to heavy workload and limited resources. With the approval, Ekterly becomes the first on-demand oral treatment for hereditary angioedema, offering a convenient alternative to injectable treatments. The life-threatening condition causes sudden, dangerous swelling in the body, including the skin, digestive tract and upper respiratory system, due to deficiency in a protein known as C1 inhibitor.