Regulatory Update
Lexicon withdrew its NDA for sotagliflozin to correct a technical issue in the NDA. Lexicon plans to resubmit the NDA in 2Q22. The FDA delayed approval of terlipressin as a treatment for hepatorenal syndrome until an inspection of the manufacturing facility can be completed. The FDA rejected oleogel-S10 for the treatment of the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa and requested additional efficacy data. The FDA agreed with the Renal Drugs Advisory Committee and rejected bardoxolone methyl, due to a lack of efficacy data to support it is use in Alport syndrome. Published Research Updates Effect of masitinib on the Expanded Disability Status Scale in patients with primary progressive multiple sclerosis. The composite clinical response in Japanese patients with non-small cell lung cancer or gastrointestinal cancer with cachexia of anamorelin. Whether adding radiotherapy to durvalumab-tremelimumab affected the overall response rates in patients with non-small-cell lung cancer that is resistant to PD-1 and PD-L1 therapy. How researchers used data from the CARTITUDE-1 trial to decrease movement and neurocognitive treatment-emergent adverse events (MNTs) with ciltacabtagene autoleucel. Check out our new lower subscription rates and don't miss out on future clinical updates Regulatory Update
The FDA approved mitapivat (Pyrukynd, Agios Pharmaceuticals), on 2/17/2022, for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will meet on 3/30/2022 to review AMX0035 for the treatment of ALS. Announced Research Updates Cytokinetics announced that in the 20-week, 276 patient, Phase II METEORIC-HF trial, treatment with omecamtiv mecarbi did not improve peak oxygen uptake during cardiopulmonary exercise testing compared to placebo in patients with HFrEF. Sage and Biogen announced that in the 15-day, 440 patient, Phase III, CORAL trial, adding zuranolone to open-label standard of care antidepressant (ADT) resulted in an 8.9 point decrease in the HAMD-17 total score at day 3 compared to a 7 point decrease with ADT alone in patients with major depressive disorder. However, the difference between zuranolone and placebo was no longer significant by day 15. BMS announced that in the 16-week, 100 patient, Phase III, VALOR-HCM trial, fewer patients treated with mavacamten elected to undergo septal reduction therapy compared to placebo in patients with symptomatic obstructive hypertrophic cardiomyopathy. Gilead announced that patients who completed the 14-day blinded portion of the CAPELLA trial were enrolled in an open-label extension where they received lenacapavir subcutaneously every six months and an optimized background regimen. After 52 weeks, 83% (30/36) achieved an undetectable viral load (< 50 copies/mL). GSK paused enrollment in three trials of its RSV vaccine candidate in pregnant patients, at the recommendation of the Independent Data Monitoring Committee, based on an observation from a routine safety assessment. Published Research Updates In the 92 patient, Phase III, REALITY trial, patients treated with apatinib achieved progression-free survival of 22.2 months compared to 4.5 months with placebo in Chinese patients with radioactive iodine–refractory differentiated thyroid cancer. A Phase II trial in adults 60 and older is not affected. Check out our new lower subscription rates and don't miss out on these clinical updates. Regulatory Update
An FDA review of sintilimab agreed with the editorial by Singha and Pazdura. The review questions whether the data from a single country is representative of the U.S. population and if use of progression-free survival instead of overall survival (OS) is acceptable. They point out that OS is a stronger endpoint and has been used for other checkpoint inhibitors that have received approval. The FDA's Oncologic Drugs Advisory Committee agreed with the FDA’s review and rejected the BLA for sintilimab plus pemetrexed and platinum-based chemotherapy for the treatment of nonsquamous non–small cell lung cancer and recommended a new clinical trial be conducted. Announced Research Updates Alzhon announced interim data after 26-weeks from 80 patents enrolled in an 84 patient, open-label, Phase II trial, where treatment with valiltramiprosate resulted in a 29% reduction in plasma p-tau181 and an improvement in the Rey Auditory Verbal Learning Test (RAVLT) in patients with early Alzheimer’s disease who carry the APOE4/4 or APOE3/4 genotype. Phathom announced that in the 42-day, 458 patient, Phase II PHALCON-NERD trial, relief of a heartburn episode was achieved within three hours with no further symptoms for 24-hours in 56% of patients treated with vonoprazan 10 mg, 60.6% with 20 mg and 70% with 40 mg compared to 27.3% with placebo in patients with non-erosive gastroesophageal reflux disease (NERD). ProQR announced that in the 12-month, 36 patient, Phase II/III, ILLUMINATE trial, treatment with sepofarsen did not improve BCVA compared to sham injection in patients with Leber’s Congenital Amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene. AZ announced that in the 1,171, open-label, Phase III HIMALAYA trial, patients treated with tremelimumab plus durvalumab achieved an objective repose rate of 20% compared to 17% with durvalumab and 5% with sorafenib in patients with hepatocellular carcinoma. Published Research Updates In the 40-week, 475 patient, Phase III SURPASS-5 trial, treatment with tirzepatide reduced HbA1C by 2.11% with 5 mg, 2.40% with 10 mg and 2.34% with 15 mg compared to a 0.86% decrease with placebo in patients with type 2 diabetes inadequately controlled with insulin glargine with or without metformin. Tirzepatide also decreased body weight more than semaglutide by 5.4 kg with 5 mg, 7.5 kg with 10 mg and 8.8 kg with 15 mg compared to a 1.6 kg decrease with placebo. In a 6-week, 150 patient, Phase III trial, the PANSS score was decreased by 20.5 with blonanserin 16 mg compared to a 10.6 point decrease with placebo in Japanese patients, 12-18 years of age, with schizophrenia. Blonanserin 8 mg did not provide an improvement compared to placebo. In a 24-week, 321 patient, Phase III trial, treatment with balovaptan did not improve the Vineland-II 2DC score compared to placebo in autistic adult patients. Check out our new lower subscription rates and don't miss out on these clinical updates New Drugs Added to Knowledgebase
Last week we added 10 new drugs to the knowledgebase:
Regulatory Update The FDA approved sutimlimab (Enjaymo, Sanofi), on 2/4/2022, to decrease the need for red blood cell transfusions due to hemolysis in cold agglutinin disease. The FDA designated CardiAMP (autologous mesenchymal stem cells), a Breakthrough Therapy for the treatment of heart failure. Harpreet Singha and Richard Pazdura, of the FDA’s Oncology Center of Excellence, published an editorial in Lancet Oncology describing how current submissions for new drugs developed in China are only including data from trials only conducted in China. They question whether the data represents a broad enough patient sample for the U.S. population. They recommend use of multiregional clinical trials, which have been used for more than 20 years, to evaluate an investigational drug submitted to the FDA. Announced Research Updates Pharming announced that in a 12-week, 31 patient, Phase II/III trial, treatment with leniolisib resulted in a reduction in lymphadenopathy lesions and an increase in B cells in patients with Activated pI3Kdelta syndrome (APDS). Vanda announced that in a 12-week, Phase III trial, treatment with tradipitant did not decrease the daily average nausea severity scores from the Gastroparesis Core Symptom Daily Diary compared to placebo in patients with gastroparesis. Published Research Updates In the 16-week, 98 patient, Phase III, THUNDER trial, treatment with fevipiprant did not improve the nasal polyp score compared to placebo in asthma patients with chronic rhinosinusitis with nasal polyps. In a 30-month, 201 patient, Phase IIb trial, 46% of patients treated with probiotic and peanut oral immunotherapy (PPOIT) and 51% of patients who received only peanut immunotherapy (OIT) achieved 8-week sustained responsiveness after 18 months of treatment compared to 5% with placebo in Australian pediatric patients with peanut allergy. There was no difference between patients that received PPOIT or OIT. Check out our new lower subscription rates and don't miss out on these clinical updates. Regulatory Update
The FDA approved faricimab (Vabysmo, Genentech/Roche), on 1/28/2022, to treat diabetic macular edema and neovascular age-related macular degeneration. The FDA approved tebentafusp (Kimmtrak, Immunocore), on 1/26/2022, for the treatment of HLA-A*02:01-positive unresectable or metastatic uveal melanoma. Tebentafusp was approved with a black boxed warning for cytokine release syndrome. Provention Bio satisfied FDA concerns for the dosing and equivalence of the AGC Biologics manufactured templizumab. A new BLA submission will also address Chemical, Manufacturing, and Controls (CMC) and product quality questions from the FDA rejection in 2021. Provention Bio plans to resubmit a BLA for templizumab in 1Q22. Incyte has discontinued its request for accelerated approval of parsaclisib for the treatment of relapsed or refractory follicular lymphoma, marginal zone lymphoma and mantle cell lymphoma, because the company felt it would be too difficult to complete required confirmatory studies to convert the accelerated approval to a full approval. The FDA paused trials evaluating the combination of magrolimab and azacitidine due to adverse reactions seen with the combination. The pause did not affect trials evaluating magrolimab alone or in combination with other chemotherapy. The FDA placed a partial hold on studies evaluating the combination of umbralisib and ublituximab in the treatment of chronic lymphocytic leukemia and non-Hodgkin’s lymphoma. The FDA designated eplontersen an Orphan Drug for the treatment of transthyretin-mediated amyloidosis. Announced Research Updates Sierra announced results of the MOMENTUM trial evaluating momelotinib in the treatment of myelofibrosis. Madrigal announced results from the MAESTRO-NAFLD-1 trial where resmetirom was evaluated for the treatment of NASH. Published Research Updates Delgocitinib was evaluated in a Phase IIb chronic hand eczema trial Plinabulin was compared to pegfilgrastim to prevent grade 4 neutropenia in the PROTECTIVE-1 trial. The BrighTNess trial evaluated the effect of adding veliparib to carboplatin in patients with triple-negative breast cancer. Check out our new lower subscription rates and don't miss out on these clinical updates. |
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