Search Results for “FDA” – Page 46 – media

Intercept restructures as Ocaliva’s NASH hopes dashed again with FDA rejection

Even after a second application attempt, Intercept’s Ocaliva wasn’t the fatty liver disease breakthrough that the company had hoped it would be. Three years after an initial rejection, the FDA once again turned down Ocaliva, or obeticholic acid, for nonalcoholic steatohepatitis (NASH) patients with stage 2 or 3 liver fibrosis, Intercept said Thursday. The denial means patients with NASH will have to wait longer for the first FDA-approved therapy and that a market estimated to be worth multiple billions of dollars still awaits a winner. For Intercept, it also means it’s time for a new direction. Intercept has decided to discontinue all NASH-related investment and restructure its operations, with the goal to save about $140 million in operating costs, the company said Friday. Measures it’s taking include winding down a long-term patient outcomes follow-up of the phase 3 REGENERATE study for Ocaliva in NASH. All R&D, commercial, medical affairs and ...
- Source: drugdu
- 102
- June 27, 2023
Amgen’s supplemental Biologics License Application for Blincyto approved by FDA

Amgen has announced the full approval by the US Food and Drug Administration (FDA) of the supplemental Biologics License Application (sBLA) for Blincyto (blinatumomab). Blincyto is used to treat adults and children with CD19-positive B-cell precursor acute lymphoblastic leukaemia (B-ALL), who are in their first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. Approved after additional data was submitted from two phase 3 studies, Blincyto’s accelerated approval is now a full approval. David Reese, executive vice president of research and development at Amgen commented that the treatment was “the first FDA-approved CD19-directed CD3 T-cell engager BiTE immunotherapy, and the first to be FDA-approved for MRD in 2018″. He added that the “full approval underscores the clinical benefit of Blincyto for people living with B-ALL”. Blincyto is a bispecific T-cell engager (BiTE) immune-oncology therapy that targets CD19 surface antigens on B cells. Helping the ...
- Source: drugdu
- 101
- June 27, 2023
After delays, Sarepta’s DMD gene therapy Elevidys finally crosses FDA finish line at $3.2M

After several delays and a narrow advisory committee vote, Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy has finally won an accelerated approval. The FDA’s approval for Elevidys comes one month behind the agency’s prior schedule and in a restricted patient population. But a win is a win for Sarepta. Specifically, the drug is approved to treat ambulatory patients ages 4 to 5 years with a confirmed mutation in the DMD gene. Sarepta is charging Elevidys, a one-time gene therapy, at a list price of $3.2 million, CEO Douglas Ingram told investors during a call Thursday. He pointed to an analysis suggesting the drug could be cost-effective at $5 million. Ingram expects the launch to take a few months to pick up, based on logistical and policy issues to address, he told investors. As opposed to an treatment outcome-based payment approach that has been adopted by other existing gene therapies to ...
- Source: drugdu
- 105
- June 26, 2023
FDA orders stores to stop selling Elf Bar disposable Vapes

The U.S. Food and Drug Administration said Thursday it has sent warning letters to 189 retailers, telling them to stop selling unauthorized tobacco products such as Elf Bar and Esco Bars. Both brands are disposable e-cigarettes sold in flavors known to appeal to kids, including bubblegum and cotton candy. A federal ban on flavored vapes that was issued in early 2020 does not cover disposable vapes. “The FDA is prepared to use all of its authorities to ensure these, and other illegal and youth-appealing products, stay out of the hands of kids,” FDA Commissioner Dr. Robert Califf said in an agency news release. “We are committed to a multi-pronged approach using regulation, compliance and enforcement action and education to protect our nation’s youth.” The warning letters were sent following a nationwide inspection blitz conducted over several weeks. In addition, two studies published in Morbidity & Mortality Weekly Report highlight concerns ...
- Source: drugdu
- 112
- June 26, 2023
FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy

By Heather McKenzie https://www.biospace.com/ Pictured: FDA sign in front of a brown brick building/Adobe Stock, Grandbrothers Sarepta’s Elevidys, approved Thursday as the first gene therapy for Duchenne muscular dystrophy, has been priced at $3.2 million per patient, making it one of the world’s most expensive medicines. The price, announced Thursday following the FDA’s decision, is second in the U.S. only to that of uniQure and CSL Behring’s hemophilia B therapy Hemgenix, approved late last year, which costs $3.5 million per patient, according to BioPharma Dive. On a conference call, Sarepta CEO Doug Ingram said the price reflected a “conservative” approach to valuing the therapy’s benefits to patients and their families, BioPharma Dive reported. Sarepta expects that discounts through Medicaid or a 340B program will make the net price of Elevidys about 20% lower than its gross cost. Original story published June 22 Sarepta’s Elevidys, approved Thursday, is now one of ...
- Source: drugdu
- 114
- June 25, 2023
Intercept winds down NASH programme after FDA pulls plug on Ocaliva

Following a negative Advisory Committee (AdCom) decision, the US Food and Drug Administration (FDA) has rejected Intercept’s Ocaliva (obeticholic acid) for the treatment of pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH) in a complete response letter (CRL) to the company. In a 23 June conference call, the company announced that it would begin the process of shutting down the REGENERATE trial (NCT02548351), with an aim to complete this process by the end of 2023. Intercept will continue to collect study data until the trial is fully terminated. Furthermore, the biotech has already begun “winding down” its NASH-related investment into research and development, commercial, medical affairs and administrative function. The company will also cut down its workforce by a third to minimise operating expenses, beginning in the coming weeks with plans to complete this by the end of 2023. Intercept will pivot its focus to Ocaliva’s use for primary biliary cholangitis ...
- Source: drugdu
- 99
- June 25, 2023
FDA approves Duchenne gene therapy with hefty $3.2m price tag

Children with a confirmed mutation of the DMD gene – responsible for causing Duchenne muscular dystrophy (DMD) – are closer to receiving the first gene therapy for the disease after the US Food and Drug Administration (FDA) approved Sarepta’s highly anticipated Elevidys (delandistrogene moxeparvovec-rokl). On a conference call following the approval, Sarepta CEO Douglas Ingram said the company has set the wholesale acquisition cost of Elevidys at $3.2m. After a narrow US Food and Drug Administration Advisory Committee (AdCom) panel vote and a delay to the Prescription Drug User Fee Act (PDUFA) date, Sarepta’s therapy crossed the approval finish line. The decision, which came through the accelerated approval pathway, is for children ages four to five with a confirmed mutation in the DMD gene. As per Sarepta, Elevidys’ price is below the cost-effectiveness range, which is estimated to be between $5m and $13m. Sarepta’s adeno-associated virus (AAV) mediated therapy delivers ...
- Source: drugdu
- 97
- June 25, 2023
Calliditas files sNDA to US FDA for TARPEYO to treat primary IgAN

Calliditas Therapeutics has submitted a supplemental new drug application (sNDA) to the US Food and Drug Administration (FDA) for complete approval of TARPEYO (budesonide). The TARPEYO delayed-release capsule is a corticosteroid indicated for reducing proteinuria to treat primary immunoglobulin A nephropathy (IgAN) in adult patients at risk of quick disease progression, generally having a urine protein-to-creatinine ratio greater than or equal to 1.5g/g. The therapy received accelerated approval based on showing a reduction in proteinuria. The submission of sNDA to the regulator was based on the findings obtained from the Phase III NefIgArd clinical trial. The multicentre, randomised, double-blind trial was designed to evaluate the safety and efficacy of TARPEYO 16 mg compared with placebo against a background of optimised RASi (renin–angiotensin system inhibitor) therapy in primary IgA adult patients. TARPEYO showed a highly statistically significant benefit in estimated glomerular filtration rate (eGFR) over a two-year period of treatment. The ...
- Source: drugdu
- 118
- June 23, 2023
SFDA approves Amarin’s VASCEPA to reduce CV risk in Saudi Arabia

The Saudi Food and Drug Authority (SFDA) has granted approval to Amarin’s VASCEPA (icosapent ethyl) capsules for reducing cardiovascular (CV) risk. VASCEPA is indicated as an adjunct to statin therapy for use in adult patients with elevated levels of triglycerides (TG). This means levels greater than or equal to 150 mg/dL. It has also been approved for use in patients at increased risk of CV events caused by at least one other CV disease risk factor and established CV disease, or who have diabetes mellitus. The therapy is the first and only medicine to receive approval from the regulator to reduce CV risk beyond cholesterol-lowering therapy in patients on high-risk statin treatment and who have increased TG levels. Amarin stated that the country’s National Heart Center (NHC) and the Saudi National Diabetes Center (SNDC) have mentioned icosapent ethyl (IPE) as an adjunct to statin therapy for CV risk reduction in ...
- Source: drugdu
- 122
- June 23, 2023
FDA Puts Mersana’s Ovarian Cancer Trials on Partial Clinical Hold

By Tristan Manalac www.biospace.com Pictured: FDA Building/courtesy of Grandbrothers/Adobe Stock The FDA has placed Mersana Therapeutics’ UP-NEXT and UPGRADE-A trials on partial clinical hold, the company announced Thursday. Both studies evaluate Mersana’s investigational antibody-drug conjugate upifitamab rilsodotin in platinum-sensitive ovarian cancer. The regulatory pause was triggered by an aggregate safety report containing information from around 560 patients who had received upifitamab rilsodotin (UpRi). Mersana’s data showed a higher rate of serious bleeding events, though most of these episodes were low-grade. Still, five cases of serious bleeding were fatal. The exact causes of bleeding are still unknown and are currently being investigated. Mersana’s stocks fell 60% in pre-market trading Thursday in response to the news. The FDA has yet to provide Mersana with a formal written communication regarding the partial clinical hold. However, the company expects the regulator to request a more comprehensive evaluation of UpRi’s safety data. Mersana is working ...
- Source: drugdu
- 100
- June 17, 2023

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