Search Results for “FDA” – Page 50 – media

FDA Delays Decision on Sarepta’s DMD Gene Therapy

By Heather McKenzie https://www.biospace.com/ Pictured: Sarepta logo on a building/courtesy of Sarepta Therapeutics Those in the Duchenne muscular dystrophy and gene therapy spaces hoping for a milestone decision by May 29 will have to wait approximately three more weeks as the FDA set a new action date of June 22 for Sarepta’s gene therapy for the neuromuscular disease. The FDA told the company Wednesday “it requires modest additional time to complete the review, including final label negotiations and postmarketing commitment discussions,” according to Sarepta’s announcement. The company went on to say that the FDA indicated it is “working toward potentially granting an accelerated approval for SRP-9001” for DMD patients aged 4-5 years. If approved, SRP-900 would be the first FDA-authorized gene therapy for DMD. Sarepta’s shares were down 14% in pre-market trading Wednesday.
- Source: drugdu
- 117
- May 26, 2023
FDA warns Sure-BioChem over quality and data issues

By Tyler Patchen News Reporter https://endpts.com/fda-warns-sure-biochem-over-quality-and-data-issues/ A warning letter has been given to a company that tests finished drug products over several quality issues. The FDA issued the warning letter to Sure-BioChem Laboratories, a Camden, NJ-based contract test laboratory that does microbial testing on products including finished drugs. Sure-BioChem was inspected late last year, but the regulator has advised the company that it still needs to make some corrections. The letter said that inspectors found that the company had failed to establish and follow procedures for some of its tests and it had not validated its use of “alternative microbial methods” used to test drug products to ensure that its methods were “Specifically, you failed to adequately establish that your microbiological testing methods can reliably detect objectionable microorganisms,” the letter said. Sure-BioChem’s methods also failed to detect Burkholderia cepacia complex (Bcc) bacteria and reported “not detected” to a client. The ...
- Source: drugdu
- 115
- May 25, 2023
Indivior’s Opvee crosses FDA finish line, adding new overdose rescue option amid raging opioid epidemic

Just two months after Emergent BioSolutions made history by winning the FDA’s blessing to sell its opioid reversal medicine over the counter, a new treatment option has arrived in Indivior’s Opvee. The nod, which covers Opvee’s use in people 12 years of age and older, marks the first approval for the medicine’s active ingredient in nasal spray form. The molecule, nalmefene hydrochloride, was first approved in the 1990s in an injectable format but was later pulled from the market in 2008. This time around, the FDA endorsed the drug based on new evidence that it can help combat rising overdose rates in the U.S. Indivior, which closed its purchase of Opiant in March and picked up the medicine in that buyout, plans to launch Opvee in the fourth quarter of 2023. The FDA’s approval comes as overdoses attributable to synthetic opioids such as fentanyl are on the rise, with more ...
- Source: drugdu
- 116
- May 25, 2023
Beta Bionics wins FDA nod to challenge Medtronic for automated insulin pump market

Dive Brief: Beta Bionics said it received 510(k) clearance from the Food and Drug Administration for its automated insulin dosing (AID) system, teeing it up to challenge Insulet, Medtronic and Tandem Diabetes Care for the Type 1 diabetes market. The system, iLet Bionic Pancreas, features an insulin pump and dosing software that combine with the Dexcom G6 continuous glucose monitor to automate the calculation and delivery of insulin doses. Beta Bionics, which showed the AID system beat standard of care last year, has differentiated its device through simplification and automation, limiting the information users need to input and preventing them from modifying their insulin doses. Dive Insight: The launch of iLet Bionic Pancreas may serve as a test of whether people with Type 1 diabetes are willing to almost totally cede control of their insulin dosing to an algorithm. Users of existing hybrid closed-loop systems enter information such as basal ...
- Source: drugdu
- 113
- May 24, 2023
AbbVie and Genmab’s blood cancer therapy granted FDA accelerated approval

AbbVie and Genmab’s blood cancer therapy, Epkinly (epcoritamab-bysp), has been granted accelerated approval by the US Food and Drug Administration (FDA). The authorisation specifically applies to adults with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who have received at least two prior lines of treatment. DLBCL is the most common type of aggressive lymphoma, with approximately 150,000 cases of the disease diagnosed globally each year. For R/R patients, several targeted therapies including T-cell mediated treatments have recently emerged, the companies said. However, single agent and ready-available or off-the-shelf treatment options are limited. Epkinly, which is being co-developed by AbbVie and Genmab as part of the companies’ oncology collaboration, is now the first and only T-cell engaging bispecific antibody for this indication. The FDA’s decision was supported by positive results from a phase 1/2 trial, in which Epkinly delivered an overall response rate of 61%, a complete response rate ...
- Source: drugdu
- 115
- May 24, 2023
Krystal Gets First FDA Approval for Redosable Gene Therapy, Rare Skin Disease

By Tristan Manalac Pictured: Blue sign outside FDA building/JHVEPhoto/Adobe Stock The FDA has approved Krystal Biotech’s topical gene therapy beremagene geperpavec-svdt, now to be marketed as Vyjuvek, for the treatment of the rare skin disease dystrophic epidermolysis bullosa, the company announced Friday. Vyjuvek’s label covers its use in dystrophic epidermolysis bullosa (DEB) patients aged six months and above bearing mutations in the COL7A1 gene. Friday’s approval makes Vyjuvek therapy the first-ever redosable gene therapy and the first DEB therapeutic to hit the market. “Until now, there have been no approved treatment for DEB in the United States, and patients, families and physicians were limited to palliative care,” Andy Orth, chief commercial officer at Krystal Biotech, said in an investor call Friday evening, adding that there are likely around 3,000 DEB patients in the U.S., of whom some 1,100 have confirmed diagnoses, according to claims analytics. The company expects to launch ...
- Source: drugdu
- 111
- May 24, 2023
Blueprint’s Ayvakit picks up long-awaited FDA approval in indolent systemic mastocytosis

In a development that’s set to change the lives of many patients, Blueprint Medicines’ Ayvakit has finally won its long-awaited FDA approval to treat indolent systemic mastocytosis (SM). The drug won its original approval in 2021 to treat the advanced form of the disease, which affects only 5% to 10% of those with SM. Now, Ayvakit can reach the majority of SM patients, who suffer from the disease’s indolent form. The larger patient pool comprises approximately 30,000 U.S. patients, but so far only about half are diagnosed, Blueprint’s chief operating officer Christy Rossi told Fierce Pharma in an interview. Indolent SM patients often end up “living their lives in a bubble,” Rossi said. Physicians can treat symptoms, which range across organ systems from skin lesions to brain fog, but until now there was no option to treat the underlying disease. Enter Ayvakit. The drug targets a mutation called KIT D816V, ...
- Source: drugdu
- 150
- May 24, 2023
Intercept’s NASH dreams may be dashed after FDA panel votes against Ocaliva’s approval bid

It looks like Intercept Pharmaceuticals’ Ocaliva still has a tough road ahead in nonalcoholic steatohepatitis (NASH) after a panel of FDA advisers voiced concerns with the company’s application package. During a one-day meeting of the FDA’s Gastrointestinal Drugs Advisory Committee, panelists voted 12 to 2—with 2 abstentions—that the benefits of Ocaliva 25 mg, also known as Obeticholic acid, or OCA, don’t outweigh the risks in NASH patients with stage 2 or 3 fibrosis. Further, in response to the question about a potential accelerated approval, panelists voted 15 to 1 against that regulatory pathway for the drug. Many of the nay-sayer’s concerns came down to Intercept’s use of a surrogate endpoint in its clinical trial. Panelists’ concerns also centered on the potential for OCA to cause drug-induced liver injury. While the FDA isn’t beholden to the advice of its advisory committees, the regulator often follows its experts’ lead. Intercept’s NASH candidate ...
- Source: drugdu
- 111
- May 23, 2023
FDA accepts Ardelyx’s NDA resubmission for XPHOZAH

The US Food and Drug Administration (FDA) has accepted Ardelyx’s resubmitted New Drug Application (NDA) for XPHOZAH (tenapanor) to control serum phosphate in adult patients with chronic kidney disease on dialysis who have had an insufficient response or intolerance to a phosphate binder therapy. The regulatory agency has determined that the NDA is a class 2 review, which leads to a six-month review period from the date of resubmission. It has set a user fee goal date of 17 October this year. The biopharmaceutical company expects XPHOZAH to be commercially available in Q4 2023 after an approval from the regulatory body. Ardelyx president and CEO Mike Raab said: “The acceptance of our NDA is a significant milestone in our journey to bring XPHOZAH to patients. We are excited about the prospect of working collaboratively with the FDA to finalise this review over the next few months. “We are now in ...
- Source: drugdu
- 101
- May 22, 2023
AbbVie’s Rinvoq scores its 7th FDA nod, this one for Crohn’s disease

In developing immunology treatment Rinvoq, AbbVie is employing the same game plan it used to make predecessor Humira a megablockbuster. And the company seems to be getting better at it.Thursday, AbbVie scored its seventh FDA approval for Rinvoq in a span of less than four years, getting the JAK inhibitor across the finish line as a treatment for Crohn’s disease. A look at Humira’s approval history shows that AbbVie has become more efficient in adding indications to its immunology blockbusters. After Humira was initially blessed in 2002, it took AbbVie 10 years to get the TNF blocker endorsed for its seventh disease type. Coincidentally, Rinvoq’s new FDA approval came hours after the U.K.’s National Institute for Health and Care Excellence signed off on the drug for Crohn’s disease. This is the second gastroenterology indication for Rinvoq after it won approval for ulcerative colitis in March of last year. Rinvoq’s first ...
- Source: drugdu
- 122
- May 21, 2023

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