Pembrolizumab (Keytruda) for Metastatic Biliary Tract Cancer
The FDA has granted approval for pembrolizumab (Keytruda) in combination with gemcitabine and cisplatin for the treatment of patients diagnosed with locally advanced unresectable or metastatic biliary tract cancer. This approval is based on the positive outcomes of the KEYNOTE-966 trial, which enrolled 1,069 patients with this condition who had not previously received systemic therapy for advanced disease. Patients receiving pembrolizumab plus chemotherapy demonstrated a significant improvement in overall survival and had an extended median overall survival compared with placebo plus chemotherapy. Approval was granted to the manufacturer of pembrolizumab, Merck. Pembrolizumab is approved for multiple cancer types and was first approved in 2014. Pembrolizumab works by releasing PD-1 pathway-mediated inhibition of the immune response, which in turn improves the anti-tumor immune response.
https://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf
Secukinumab (Cosentyx) for Moderate-to-Severe Hidradenitis Suppurativa
The FDA has granted approval for secukinumab (Cosentyx) as a treatment option for adults with moderate-to-severe hidradenitis suppurativa (HS). This approval makes secukinumab the first and only FDA-approved interleukin (IL)-17A inhibitor for HS, a condition affecting approximately 1% of the global population. The approval for HS is based on data from the pivotal phase 3 SUNSHINE and SUNRISE trials, which collectively enrolled over 1,000 HS patients from 40 countries. A significantly higher proportion of patients achieved a response when treated with secukinumab compared to placebo in both SUNSHINE and SUNRISE trials at week 16. Additionally, an exploratory analysis extending to 52 weeks showed further improvements in response following either dose regimen. Secukinumab, manufactured by Novartis, was first approved in 2015 and is also indicated for moderate-to-severe plaque psoriasis, psoriatic arthritis, and ankylosing spondylitis.
https://www.novartis.com/us-en/sites/novartis_us/files/cosentyx.pdf
Ustekinumab-auub (Wezlana) Biosimilar for Multiple Inflammatory Diseases
The FDA has granted approval for Wezlana (ustekinumab-auub) as a biosimilar that is interchangeable with Stelara for the treatment of multiple inflammatory diseases, including Crohn’s disease and psoriatic arthritis. According to the FDA’s news release, the approval of Wezlana is based on a comprehensive review of scientific evidence demonstrating that it is highly similar to Stelara and that there are no clinically meaningful differences between the two products in terms of safety, purity and potency. Approval of Wezlana was granted to Amgen, Inc.
Vonoprazan (Voquezna) for Erosive Esophagitis and GERD
The FDA has approved vonoprazan (Voquezna) for the treatment of erosive esophagitis, also called erosive gastroesophageal reflux disease (GERD). Vonoprazan, a first-in-class oral potassium-competitive acid blocker (PCAB), provides more potent inhibition of gastric acid than proton pump inhibitors (PPIs) and is seen as a potential alternative. Approval is based on the phase 3 PHALCON-EE study involving 1024 patients in the US and Europe, comparing vonoprazan to lansoprazole (Prevacid) for GERD management. Vonoprazan 20 mg achieved noninferiority to lansoprazole 30 mg for complete healing by week 8. Adverse event rates were similar to lansoprazole. Vonoprazan, manufactured by Phathom Pharmaceuticals, is expected to be available in the US next month. This week’s approval follows an approval last week of the Voquezna Triple Pak (vonoprazan, amoxicillin, clarithromycin) and the Voquezna Dual Pak (vonoprazan, amoxicillin) for the treatment of Helicobacter pylori infection in adults.
https://www.phathompharma.com/wp-content/uploads/VOQUEZNA-tablets-Prescriber-Information.pdf
Abatacept (Orencia) In Juvenile Psoriatic Arthritis
The FDA has approved an expanded indication for abatacept (Orencia) for the treatment of psoriatic arthritis in pediatric patients 2 years of age and older. Abatacept, manufactured by Bristol Myers Squibb was first approved in 2005 for the treatment of moderate to severe rheumatoid arthritis and was approved for treating active PsA in adults in 2017. https://packageinserts.bms.com/pi/pi_orencia.pdf
Exa-cel CRISPR for Sickle Cell
It’s not an approval yet, but an FDA advisory panel concluded last week that a CRISPR approach for sickle cell disease is safe enough for clinical use and may be approved as early as December 8. Exagamglogene autotemcel (exa-cel), manufactured by CRISPR Therapeutics and Vertex, would be the first approved therapeutic based on CRISPR gene-editing technology. Currently there are over 250 CRISPR-based therapies in clinical trials worldwide, targeting a wide range of diseases, including several cancer types, rare diseases, and infectious diseases. The approach involves removal of cells from patients followed by gene editing so that, in the case of sickle cell disease, the cells produce high levels of fetal hemoglobin (hemoglobin F) in red blood cells, then infusion of the modified cells back into patients. This then restores normal red blood cell function. While not a cure, the hope is that exa-cel will be a one-time treatment that will alleviate symptoms of sickle cell disease for a lifetime. In a study involving 30 patients, exa-cel alleviated symptoms in 29 patients followed for at least 18 months and did not appear to cause any serious short-term safety issues. Exa-cell is being evaluated in the CLIMB 141 AND 151 Phase 3 clinical trials in sickle cell disease and beta-thalassemia, respectively.
https://www.fda.gov/media/173414/download
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