Eli Lilly has announced positive top-line results from a late-stage study of its targeted therapy Retevmo (selpercatinib) versus the current first-line standard of care in certain non-small cell lung cancer (NSCLC) patients. The phase 3 LIBRETTO-431 trial has been evaluating Retevmo versus platinum-based chemotherapy plus pemetrexed with or without Merck’s Keytruda (pembrolizumab) as an initial treatment for adults with rearranged during transfection (RET) fusion-positive advanced or metastatic NSCLC. A pre-specified interim efficacy analysis showed that the study met its primary endpoint, with Lilly’s therapy demonstrating a statistically significant and clinically meaningful improvement in progression-free survival. As the company outlined, LIBRETTO-431 is the first randomised study to compare the safety and effectiveness of a targeted therapy to a PD-1 inhibitor plus chemotherapy in cancer patients bearing a specific biomarker. David Hyman, chief medical officer at Loxo@Lilly, said: “The LIBRETTO-431 trial aims to answer an important question about the selection of initial ...
The National Institute for Health and Care Excellence (NICE) has issued a draft Appraisal Consultation Document that does not recommend CSL Behring’s Hemgenix (etranacogene dezaparvovec) gene therapy for haemophilia B. The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors. In its guidance, NICE’s evaluation committee said that while there is clear evidence that the therapy reduces the number of bleeding episodes haemophilia B patients have each year, there is not enough evidence on how well it works in the long term, meaning cost-effectiveness estimates are “uncertain”. It also cited problems with the indirect comparison between the gene therapy and factor IX replacement therapies. Haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting factor IX, a protein needed to produce blood clots to stop bleeding. Patients ...
More than 2.4 million people in the United States use warfarin to keep their blood from clotting after a heart attack, stroke or other serious thromboembolic complication. But using a blood thinner carries risks of its own as patients become prone to bleeding, particularly during urgent surgery or other invasive procedures. To combat the risk, Swiss plasma specialist Octapharma has developed a treatment that rapidly restores the blood’s ability to coagulate. On Wednesday, the FDA signed off on the company’s Balfaxar (prothrombin complex concentrate, human-lans). Already marketed as Octaplex in Europe and Canada, Balfaxar is for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonists such as warfarin. Balfaxar, which is a lyophilized powder for reconstitution, is provided with sterile water for injection by way of a new transfer device called Nextaro. The drug earned approval based on a phase 3 trial that ...
Swiss-based Novartis announced that the US District Court for the District of Delaware has invalidated the patent covering its cardiac drug Entresto, which expires on 15 July 2025 with the associated paediatric exclusivity. The company said it will now appeal to the US Court of Appeals for the Federal Circuit (CAFC) to further pursue the efforts to validate the combination patent around Entresto. A combination of sacubitril and valsartan, Entresto was first approved by the US Food and Drug Administration (FDA) to treat heart failure with reduced ejection fraction in July 2015. It is also approved to treat symptomatic heart failure with systemic left ventricular systolic dysfunction in pediatric patients. Apart from the patent which was adjudicated by the District Court, additional patents for the combinations of sacubitril and valsartan, including the drug forms and dosages, are listed to expire from November 2026 to May 2036. ...
Boehringer Ingelheim’s new autoinjector pen administering Cyltezo (adalimumab-adbm) is now available to patients living with chronic inflammatory diseases in the US. The Cyltezo pen autoinjector, which is a biosimilar to AbbVie’s blockbuster Humira (adalimumab), received approval from the US Food and Drug Administration (FDA) in May 2023. Boehringer Ingelheim originally received FDA approval for the drug in 2017, and then further approval as an interchangeable biosimilar in 2021. The 40mg/0.8ml pre-filled Cyltezo Pen will be offered in two, four and six-pack options. AbbVie has enjoyed a prosperous monopoly of the adalimumab market with Humira generating $21.2bn in global sales in 2022. With several biosimilars likely to flood the market in 2023 and the company losing exclusivity, AbbVie said it expects its sales of the drug to decline 37% in 2023. Challengers to Humira are now vying for market share. Organon and Samsung Bioepsis launched an autoinjector pen on 1 July, ...
The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) for Alvotech’s Humira biosimilar AVT02, further delaying the company’s plans to launch the drug in a landmark year. The agency rejected the drug’s biologics licence application (BLA) over deficiencies seen at one of the company’s manufacturing facilities in Reykjavik, Iceland, according to Alvotech’s press release on 28 June. While the FDA did not make a note of any other deficiencies in the BLA, issues related to the Reykjavik facility need to be resolved to facilitate the drug’s potential approval. This marks the second time that the FDA has rejected AVT02’s BLA this year. In April, the regulator issued a CRL to Alvotech, which also noted the deficiencies seen in the company’s Reykjavik plant. Prior to that, the company received a CRL from the FDA over deficiencies in the plant in September 2022. Alvotech plans to resubmit ...
After an initial rejection in 2020 and a review delay earlier this year, BioMarin’s Roctavian has finally got the FDA go-ahead to introduce a gene therapy for a not-so-rare disorder.The FDA has cleared Roctavian as a one-time therapy for adults with severe hemophilia A, BioMarin said Thursday. The news comes seven months after the FDA greenlighted CSL Behring’s hemophilia B gene therapy Hemgenix, which bears a list price of $3.5 million and is currently the most expensive drug in the world. BioMarin is pricing Roctavian at a wholesale acquisition cost of $2.9 million, BioMarin’s chief commercial officer Jeff Ajer said during a call Thursday. It’s also setting up an outcomes-based warranty program. The warranty will reimburse government and commercial payers up to the full cost if Roctavian doesn’t live up to its treatment expectations. Partial reimbursement will be granted if an individual loses response to the therapy in the first four ...
By Elaine Chen STAT – Reporting from the frontiers of health and medicine Liver illustration HYACINTH EMPINADO/STAT SAN DIEGO — The American Diabetes Association said Sunday that all adults with type 2 diabetes or prediabetes should be screened for nonalcoholic fatty liver disease, an increasingly prevalent condition that can lead to serious liver damage. There are no approved medications for the disease, but among available diabetes drugs, the ADA singled out GLP-1 treatments as an option doctors could consider, according to recommendations published during the annual ADA conference. GLP-1 treatments, such as Ozempic and Mounjaro, are a class of drugs that have grown widely popular for their efficacy not only in lowering blood sugar, but also cutting weight. Drugmakers have started to study them in liver disease, and while some trials have shown they may offer some benefits, they haven’t yet been shown to improve harmful liver scarring. The ADA ...
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 ...
Fever was found to be the most common non-respiratory feature of infection with SARS-CoV-2, the virus that causes COVID-19, according to research published at the ATS 2023 International Conference. The finding held true regardless of which COVID variant patients had, and whether or not they were fully vaccinated or not fully vaccinated. The researchers, who also looked at mortality risk, found that patients who were not fully vaccinated had a higher risk of dying when infected with either the Omicron or Delta variant. The study was based on the examination of the University of California Health Covid Research Data Set’s (UC CORDS) medical records of 63,454 patients who had been treated in a University of California medical center for COVID-19. The scientists applied statistical tests to determine the relationship between non-respiratory features, vaccination status and differences in mortality between infection with the Omicron and Delta variants. We determined that we ...
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