BMS immunotherapy combination gets two FDA nods for GI cancer in one week

In regulatory decisions over the past week, first-line treatment of two types of gastrointestinal cancer now allows pairing of first-line treatments for both types of gastrointestinal cancer.

Drugs from Opdivo and Yervoy fall into the category of drugs called checkpoint inhibitors. On April 8, the FDA approved the use of this drug combination for adults and children aged 12 and older whose colorectal cancer has metastasized or cannot be removed surgically. Cancer must also be high microsatellite instability (MSI-H) or mismatch repair defects (DMMR), which cites biomarkers that demonstrate DNA stability in tumors.

The regulatory submission is based on the results of the Open Label Phase 3 test that compared the Opdivo/Yervoy combination with OPDIVO or chemotherapy alone. The results show that the combination of immunotherapy results in a 38% reduction in the risk of disease progression or death compared to OPDIVO monotherapy, while both in front-line settings by 79% compared to chemotherapy. The study is undergoing evaluation of secondary measures, including overall survival. BMS said it will continue to work with research researchers to present these data and future long-term follow-up results.

On April 11, the FDA approved the combination of Opdivo and Yervoy for the treatment of advanced hepatocellular carcinoma. In the critical test in this indication, the drug combination resulted in an overall survival of 23.7 months. The median overall survival was 20.6 months in the comparison arm when the patient received the care medications sorafenib or ritinib. Over three years, the overall survival rate of this drug combination was 38%, while the overall survival rate of the comparative arm was 24%. The FDA has obtained the approval of Opdivo Plus Yervoy in hepatocellular carcinoma, which is the European Commission’s approval of drug combinations in this direction.

There is a lot of news on regulation. Here is a review of other recent highlights:

New and expanded regulatory approvals

– Argenx Drug Vyvgart Hytrulo has obtained additional approval for the product through a prefilled syringe. The drug is provided in vials and has previously been approved by the FDA, which has obtained chronic inflammatory demyelinated polyneuropathy of autoimmune conditions and summarizes myostria. Analysts say preperfusion syringes bring more convenient dose options to patients, while providing Argenx with the opportunity to gain more market share by penetrating early treatment options.

– Amgen's Uplizna has expanded its tags to include the treatment of immunoglobulin G4-related diseases (IgG4-RD), Immune-mediated inflammatory disease that may affect multiple organs. Regulatory decisions make intravenous antibodies the first FDA-approved treatment for this rare and chronic disease. Uplizna was first approved in 2020 to treat neuromacular diseases with Optica Speprum disorder.

– Bayer's Viktrakvi has now been fully approved by the FDA for the treatment of NTRK gene fusion-positive solid tumors in adults and adults and children without known acquired drug resistance mutations. The drug received accelerated approval for this indication in 2018.

– Astrazeneca and Daiichi Sankyo welcome the EU's recognition of Datroway as a treatment for HR-positive and HER2-negative metastatic breast cancer. Datroway received FDA approval for the same direction in January. The drug is an antibody drug conjugate designed to target the oncoprotein Trop2.

– Novartis Drug Atrasentan, brand Vanrafia, was awarded accelerated approval as a new approach to immunoglobulin A nephropathy (IGAN) for kidney disease. The drug is an oral small molecule designed to block the endothelin A receptor. This is one of two IGAN nephropathy drugs that were obtained through the $3.2 billion acquisition of Chinook Therapeutics in 2023. Novartis’s internal Igan Research produced the complement inhibitor Fabhalta, which received accelerated approval in this sign last summer.

– The Novartis drug claims extend its label to include C3 glomerulopathy, making the pill twice a day the first approved treatment for this rare kidney disease. Fabhalta is a small molecule designed to block complement protein C3. It was first approved in 2023 to treat rare nocturnal nocturnal hemoglobinuria.

– Sanofi has been approved by FDA Fitusiran, brand Qfitlia, as a treatment for hemophilia and B. The drug uses RNA to interfere with lower AT, a protein that inhibits blood clotting. The approval covers treatment for adults aged 12 and older and children with inhibitors, which may develop in hemophilia patients, making it harder to control bleeding. In contrast, the new Pfizer drug hymn is only used in patients with hemophilia A and B without inhibitors, while SANOFI's ALHEMO is only approved for patients with hemophilia A and B who are only used in patients with inhibitors.

– Blocky AstraZeneca Cancer Immunotherapy has been approved by the EU for the treatment of limited stages of small cell lung cancer. The new approval is based on the results of Phase 3, indicating a 27% reduction in the risk of death compared to placebo. The FDA approved this sign of Imfinzi in December last year.

– imfinzi also adds perioperative treatment for muscle-invasive bladder cancer to its FDA-approved list of instructions. The latest FDA nod covers the use of the drug in combination with chemotherapy to remove the bladder before surgery, followed by Imfinzi as an auxiliary monotherapy. In key studies supporting this new sign, the results show that compared with neoadjuvant chemotherapy, the risk of cancer recurrence is 32% lower and the risk of death is 25%.

– The European Commission approved the Pfizer vaccine Abrysvo to prevent respiratory syncytial virus (RSV). Regulatory decisions have extended the approval of the vaccine to adults aged 18 to 59. European regulators initially approved the vaccine in 2023 to prevent lower respiratory tract disease in adults aged 60 and over. Abrysvo won FDA approval the same year.

– FDA has expanded approval of Novartis' Pluvicto to include earlier treatments before use as anti-androgenic drugs and chemotherapy – for PSMA-positive metastatic inhibitory persistent drug-resistant prostate cancer (MCRPC). Pluvicto was initially approved in 2022 for PSMA-positive MCRPCs that have been treated with anti-androgenic drugs and chemotherapy. Novartis said this expansion is important because about half of the patients live less than enough to receive second-line treatment.

– The long-term cancer drug Cabometyx extends its FDA-approved use to advanced cases of pancreatic neuroendocrine tumors (PNET) and extrapancreatic neuroendocrine tumors (EPNET). The approval covers adults and children aged 12 and over. Cabometyx is a small molecule designed to block enzymes that support cancer growth. The drug was first approved for advanced renal cell carcinoma in 2016.

– GSK landed FDA-approved Blujepa, an oral antibiotic used to treat simple urinary tract infections. Although drugs are already available for these infections, they can be resistant to treatment. Blujepa aims to block bacterial DNA replication from two enzyme targets, and the approach aims to reduce the potential of drug resistance.

– European Commission approved Capvaxive, a pneumococcal vaccine developed by Merck To prevent 21 S. pneumoniae serotypes that cause most invasive pneumococcal diseases, including 8 that are not covered by any other available pneumococcal vaccines. European regulatory decisions cover adults aged 18 and older. Capvaxive received FDA approval last July.

– Bristol Myers' squibb's Breyanzi has been approved by the European Commission for relapse or refractory follicle lymphoma. This sign was accelerated approval from the FDA in this direction last May.

– Geron's Rytelo has been approved by the European Commission to treat transfusion-dependent anemia caused by low-risk bone marrow dysplasia syndromea progressive blood cancer. Intravenous drugs were the first telomerase inhibitor to enter the market. Rytelo received FDA approval last June.

– Neffy is an adrenaline nasal spray, ARS drug developed its FDA approval for treating allergic reactions to insect bites, medications and foods Included patients weighing 15 to 30 kg (about 33 to 60 lbs). The initial approval of the product last year covered its use in patients weighing 30 kg or more.

– Roche subsidiary Genentech has received FDA approval for tenecteplase, brand TNKase, as a treatment for acute ischemic stroke in adults. Genentech said TNKase is the first batch of stroke medicines approved by the FDA in nearly 30 years. This is the second approval instruction for clot damage drugs. The FDA approved the treatment of acute myocardial infarction in 2000.

– Neurotechnical drug cell and gene therapy administered by surgical implants has been approved by FDA for the treatment of type 2 macular telangectia. The therapy, the brand name Endelto, is the first approved treatment for this rare vision disorder.

Regulatory setbacks

– FDA missed the April 1 target date for regulatory decisions for the Novavax Covid-19 vaccine. Politico reported that the holding was due to intervention by the FDA's principal deputy commissioner Sara Brenner, who requested more data. Novavax said it has responded to all FDA requests for information and believes that the application has been prepared for approval based on submitted Phase 3 data.

Novavax’s protein-based vaccine was originally granted an FDA Emergency Use Authorization in 2022, a status that remained under revised authorization in 2023 and last year, both involving new formulations covering universal variants at the time. Comprehensive FDA approval will put the vaccine on par with the approved Messenger RNA vaccines sold by Pfizer and ModernA. Novavax's Covid-19 vaccine will be commercially available with Sanofi under a partner announced last year.

The drug giant said in a letter to the World Duchin that Roche stopped European clinical testing by the European Bureau of Muscular Dystrophy Gene Therapy. The move comes after Sarepta Therapeutics' recent reports of deaths in patients with liver failure-related diseases. Roach said the European Medicines Agency requires clinical holdings of four studies until the cause of death is investigated. Roche owns Elevidys outside the United States under a 2019 agreement with Sarepta

– For the second time, the FDA rejected Aldeyra Therapeutics Drug Reproxalap as a treatment for dry eye. The agency rejected the drug's application in 2023. According to Aldeyra, the FDA's latest full reply letter said that the new drug application failed to prove its efficacy. Regulators require the company to conduct at least one more clinical trial. The focus raised by the FDA is on differences in baseline scores, which may have affected the interpretation of trial results. Aldeyra said the results of two ongoing studies are expected to be conducted later this quarter, which may pave the way for the resubmission of the application.

– The European Medicines Agency rejects marketing authorization from Eli Lilly Alzheimer's Demand Drug Kisunla. Safety is the main cause of citation, especially brain inflammation and bleeding, which are known complication risks associated with all drugs in the same class of Alzheimer's drugs. The agency said the drug's benefits are not enough to outweigh potential fatal safety risks. The FDA approved Kisunla in July last year.

– In other drug news about Alzheimer's, Esey once again failed to convince Australia's drug regulator to register Leqembi. The pharmaceutical company has asked the Treatment Goods Administration (TGA) to reconsider its October 2024 decision not to register the drug in Australia. Eisai’s reconsideration requirement covers narrower indications, but Eisai said TGA does not agree to establish safety for these patients.

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