Search Results for “FDA” – Page 53 – media

Protalix, Chiesi challenge Sanofi with EU approval for Fabry disease med as FDA decision looms

Protalix BioTherapeutics and Chiesi Farmaceutici have endured a rejection from the FDA and conducted multiple readouts of a phase 3 trial of their Fabry disease candidate. After many stops and starts, the companies have finally scored a marketing approval in Europe for PRX-102 (pegunigalsidase alfa), an enzyme replacement therapy for the rare, genetic, progressive disorder, which causes an accumulation of fatty deposits in the lysosomes and strikes roughly 1 in 50,000 people. The authorization is based on results from a clinical program that has tested PRX-102 in more than 140 patients with up to 7.5 years of treatment. A head-to-head trial pitting PRX-102 against Sanofi’s Fabrazyme (agalsidase beta) demonstrated noninferior efficacy in controlling the kidney disease that accompanies the disorder. Fabrazyme, which was approved 20 years ago, is the longtime dominant drug in the Fabry disease market, generating sales of 938 million euros ($986 million) last year. Amicus Therapeutics of Philadelphia, which earned an FDA approval for its ...
- Source: drugdu
- 178
- May 8, 2023
Formus Labs’ Hip Surgery Planning Software Expands with FDA Clearance

In order to carry out a seamless joint replacement, surgeons must create a customized preoperative plan for each patient. However, surgeons having to perform multiple operations per day are often too busy to dedicate adequate time to this step. Formus Labs, a New Zealand-based medical technology startup, is on a mission to make the preoperative planning process much quicker. On Wednesday, the company received 510(k) clearance from the FDA for Formus Hip, its fully automated 3D software for hip replacement preoperative planning. The company announced its plans to expand into the U.S. last year. Wednesday’s FDA clearance represents a major milestone, as Formus can now begin to sell its product to surgeons and healthcare providers across the country. Founded in 2017, Formus seeks to solve the problem of patient dissatisfaction and unsustainable costs in joint replacement surgery, said CEO Ju Zhang in a recent interview. “Our approach to solving this ...
- Source: drugdu
- 122
- May 7, 2023
FDA issues new draft guidance on decentralised clinical trials

The US Food and Drug Administration (FDA) has issued new draft guidance on the implementation of decentralised clinical trials (DCTs), in which some or all trial-related activities occur at locations other than traditional investigation sites. This could include carrying out clinical tests at a local facility rather than a research medical centre, or conducting follow-up visits in the participants’ homes using telemedicine. The 16-page document provides recommendations for sponsors, investigators and other stakeholders on the use of these trials, which the agency expects’ will play an important role in addressing public health needs’. ‘By reducing barriers to participation, we expect that DCTs will increase the breadth and diversity of participants in clinical trials and improve accessibility for those with rare diseases or mobility challenges,’ the FDA said in a statement. The guidance covers recommendations on topics such as DCT design, the use of digital health technologies for remote patient monitoring, ...
- Source: drugdu
- 120
- May 6, 2023
Seres’s Bacteria in a Pill Becomes First FDA-Approved Oral Microbiome Therapy

Recurrent Clostridioides difficile infection can be treated with fecal microbiota transplants. FDA approval of Seres Therapeutics’ Vowst makes it the first oral microbiome therapy. A capsule filled with bacteria is now FDA approved for treating a serious and potentially deadly type of gut infection, making the Seres Therapeutics drug the first oral microbiome therapy to receive the regulatory go-ahead for patients. The infection is caused by Clostridioides difficile (C. difficile or C. diff), a bacterium that’s part of the diverse microbial community in the intestines. When the gut microbiome is disrupted, C. diff can proliferate, releasing toxins that cause frequent diarrhea, abdominal pain, and fever. In severe cases, the infection leads to organ failure and death. Antibiotics can treat C. diff infection, but these bacteria can develop resistance, leading to a recurrence of the infection. When that happens, patients have limited treatment options. According to Centers for Disease Control and Prevention data cited by Seres, about 156,000 cases of ...
- Source: drugdu
- 169
- May 2, 2023
FDA clears Pfizer’s pneumococcal vaccine for infants and children

Pfizer on Thursday gained U.S. approval of its newest pneumococcal conjugate vaccine for use in children and infants, helping it compete with a rival shot from Merck & Co. that won a similar OK last year. Pfizer’s vaccine, called Prevnar 20, is designed to protect against infection by 20 strains of the bacteria — seven more than the company’s prior Prevnar 13 version. It has been approved in adults since 2021. Last July, Merck won approval in children of its Vaxneuvance vaccine, which protects against 15 strains. Both companies could soon face more competition as the market has sparked interest from other drugmakers. While vaccines for the bacterial infection are widely available, thousands of cases still lead to hospitalization and death in the U.S. each year. In response, Pfizer, Merck and others have worked to develop new shots that offer better protection, particularly against strains that weren’t ...
- Source: drugdu
- 227
- May 1, 2023
Medicare will cover Leqembi for all patients if FDA approves drug, CMS chief says

Medicare will cover the new Alzheimer’s treatment Leqembi for all patients eligible under the medication’s label if the Food and Drug Administration fully approves the drug in July, a federal official told members of Congress on Wednesday. The official, Chiquita Brooks-LaSure testified before Congress Wednesday for the first time since her confirmation as administrator of the Centers for Medicare and Medicaid Services. Brooks-LaSure faced pointed criticism from Democrat and Republican members of the House Subcommittee on Health over Medicare’s controversial coverage policy for new Alzheimer’s treatments. The Food and Drug Administration approved Leqembi, which is a collaboration by Biogen and Eisai antibody treatment, on an expedited basis in January. Twice-monthly intravenous infusions of the drug, generically known as lecanemab, have shown promise in slowing the progression of early Alzheimer’s disease. But Medicare, which primarily provides health coverage to senior citizens, currently will only cover the ...
- Source: drugdu
- 116
- May 1, 2023
FDA clears Pfizer’s pneumococcal vaccine for infants and children

Delilah AlvaradoAssociate Editor A general view of the Pfizer Headquarters sign on November 10, 2020 in Tadworth, England. Dan Kitwood via Getty Images Dive Brief: Pfizer on Thursday gained U.S. approval of its newest pneumococcal conjugate vaccine for use in children and infants, helping it compete with a rival shot from Merck & Co. that won a similar OK last year. Pfizer’s vaccine, called Prevnar 20, is designed to protect against infection by 20 strains of the bacteria — seven more than the company’s prior Prevnar 13 version. It has been approved in adults since 2021. Last July, Merck won approval in children of its Vaxneuvance vaccine, which protects against 15 strains. Both companies could soon face more competition as the market has sparked interest from other drugmakers. Dive Insight: While vaccines for the bacterial infection are widely available, thousands of cases still lead to hospitalization and death in the ...
- Source: drugdu
- 155
- April 30, 2023
BrainStorm files ALS drug application over FDA protest

Ned Pagliarulo Lead Editor A sign for the Food And Drug Administration is seen outside of the headquarters on July 20, 2020 in White Oak, Maryland. Sarah Silbiger via Getty Images A yearslong disagreement between the Food and Drug Administration and a developer of an experimental medicine for ALS will get a public hearing sometime in the future, setting up another high-profile debate of what amount of evidence merits an approval for the progressive and fatal disease. On Monday, BrainStorm Cell Therapeutics said the agency will convene a panel of outside experts to review its medicine, a personalized stem cell treatment called NurOwn. The planned meeting, which has not yet been scheduled, is the result of BrainStorm taking the rare step of requesting its approval application be filed over the FDA’s protest. Last November, the regulator refused to review BrainStorm’s application, which was based on a late-stage clinical trial that failed to meet its main goal. ...
- Source: drugdu
- 169
- April 28, 2023
FDA awards Biogen’s ALS drug Qalsody an accelerated approval, following its experts’ feedback this time

The FDA has approved another Biogen drug for a neurological disorder. And, unlike the controversial go-ahead for Aduhelm, the agency’s neuroscience division didn’t go against its external advisers’ advice this time. Tuesday, the FDA granted an accelerated approval to Biogen and Ionis’ tofersen to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene. The drug will be sold under the brand name Qalsody. The move follows the recommendation from an FDA advisory committee in March. At that time, nine experts agreed that Qalsody’s ability to reduce plasma neurofilament light (NfL), a biomarker of nerve injury and neurodegeneration, provides enough basis for a conditional approval. However, thanks to a phase 3 trial flop, the panel voted against full approval. SOD1-ALS is a small subset of ALS, affecting fewer than 500 patients in the U.S. and about 2% of all 168,000 ALS patients ...
- Source: drugdu
- 262
- April 27, 2023
After delay, Bluebird submits sickle cell gene therapy for FDA approval

Bluebird bio said Monday it has submitted its lovo-cel gene therapy for sickle cell disease for Food and Drug Administration approval, ending a brief delay as it awaited feedback from the agency on manufacturing. The Massachusetts-based drugmaker missed its goal to submit an application by the end of March, allowing Vertex Pharmaceuticals and CRISPR Therapeutics to jump ahead with an approval application for a competing therapy. The agency now has until late June to decide whether to accept Bluebird’s application. The company is seeking a “priority,” or expedited, review, which if granted would shorten the review from 10 months to six. Bluebird is planning for an expedited review and, should an approval be granted, an early 2024 commercial launch. Lovo-cel is viewed by analysts as Bluebird’s best chance for business success because many more people in the U.S. have sickle cell disease than the rarer conditions ...
- Source: drugdu
- 163
- April 26, 2023

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