We evaluate over 1,000 data feeds regarding investigational drugs to keep information for 630 drugs in our database current, so you can stay up to date. Here is a summary of the significant developments from last week.
The FDA approved Medivation’s talazoparib (Talzenna) on October 16, nearly a month ahead of its PDUFA Date, for the treatment of advanced BCRA-positive breast cancer. We list two published studies for Talazoparib. Try us for a month to review talazoparib’s published studies and learn about an additional twenty breast cancer investigational drugs in the Pharmaceutical Pipeline Tracker. Click on this link for one-month’s access to the Pharmaceutical Pipeline Tracker @ $115 with no long-term commitment: https://www.prescriberight.com/subscription-offers.html
An FDA Advisory Committee voted 10-0 to recommend prucalopride tablets for the treatment of adults with chronic idiopathic constipation. In a review of Shire and Janssen’s prucalopride, the FDA noted that despite the drug being available in Europe since 2009, there have been no controlled trials lasting at least 12 months. In another FDA review, an integrated safety analysis of 2552 patients, the most common ADR in prucalopride trials were gastrointestinal disorders including nausea, diarrhea, abdominal pain, and headache with similar cardiovascular events compared to placebo. A final decision on prucalopride is expected in December. The drug does not have a PDUFA Date nor any Priority Designation.
Alnylam is planning to begin a rolling NDA for givosiran by the end of the year with completion in mid-2019, after full results from a Phase III trial are available. Givosiran has Orphan Drug and Breakthrough Therapy priority designations.
MedDay announced that in a 12-month, 220 patient, open label trial, treatment with high dose biotin improved the Expanded Disability Status Scale (EDSS) and timed 25-foot walk (T25W) from baseline to one year in patients with progressive multiple sclerosis or secondary progressive multiple sclerosis. CHMP did not recommend approval of high dose biotin for MS due to a lack of clinical evidence.
Sarepta licensed U.S. and non-European marketing rights for LYS-SAF302 from Lysogene. There is one citation in the Pharmaceutical Pipeline Tracker. There are twenty additional gene therapy investigational drugs in our Knowledgebase. Try us for a month to review Lysogene’s published finding and some of the other over 800 additional clinical studies in our Knowledgebase. Click on this link for one month’s access to the Pharmaceutical Pipeline tracker @ $115 with no long-term commitment: https://www.prescriberight.com/subscription-offers.html Search for gene therapy under the Indication Search button to see the list of twenty-one gene therapy investigational drugs and review each drug individually.
Celgene announced that in a Phase III trial, treatment with ozanimod resulted in a lower relapse rate and fewer brain MRI lesions compared to interferon beta-1a in patients with relapsing multiple sclerosis. The company also announced that in a pooled analysis of the phase III trial and another 24-month trial, ozanimod reduced the annual relapse rates by 43% with a 1 mg dose and 26% with a 0.5 mg dose compared to interferon beta-1a in 2,666 patients with relapsing multiple sclerosis.
Mylan and Theravance announced that in a 28-day, 207 patient, Phase III trial, nebulized revefenacin had similar improvements in trough FEV1 and trough FVC to MDI tiotropium in patients with COPD. The drug has a Nov 13, 2018, PDUFA Date. In a pooled analysis of three Phase II trials and a Phase I trial, nebulized revefenacin did not prolong QT interval nor increase risk of major adverse cardiac events (MACE).
AZ announced that in a Phase III trial, the addition of selumetinib to radioactive iodine did not improve complete remission compared to radioactive iodine alone in patients with nonmetastatic differentiated thyroid cancer that were post total thyroidectomy. Selumetinib was granted an Orphan Drug Priority Designation by the FDA.
Eisai announced that in a Phase III trial, lemborexant reduced sleep latency compared to placebo. In a Phase III trial the most common ADR with lemborexant were headache, somnolence and influenza.
Allergan announced the most common ADR with ubrogepant in a 1,230-patient open label extension safety trial were nasopharyngitis, upper respiratory tract infection, sinusitis, urinary tract infection, and influenza. In a second safety trial, involving 516 patients, the most common ADR were headache, oropharyngeal pain, and nasopharyngitis. No hepatic safety concerns were noted in either safety trial. Allergan plans to submit an NDA for ubrogepant in 1Q19, for migraine prophylaxis. Four additional drugs of the eight migraine drugs still in development are being studied for migraine prophylaxis.
The New England Journal of Medicine published two trials of cystic fibrosis transmembrane conductance regulator (CFTR) correctors being developed by Vertex. In a Phase II study, VX-445 in combination with tezacaftor/ivacaftor increased FEV1 by 8 to 14% compared to no improvement with placebo. In the second Phase II study, VX-659 in combination with tezacaftor/ivacaftor increased FEV1 by 10 to 13% compared to 0.4% improvement with placebo. In both Phase II trials for VX-445 or VX-659, ADR were mostly mild to moderate with no dose-limiting side effects or toxic effects. Researchers estimate the combination of either VX-445 or VX-659 in combination with tezacaftor/ivacaftor could treat the underlying cause of CFTR in 90% of cystic fibrosis patients. Vertex has three ongoing trials for each drug in combination with tezacaftor/ivacaftor for the treatment of cystic fibrosis.
Novartis has the ongoing Phase III study evaluating intravenous AVXS-101 in spinal muscular atrophy Type 1, 2 & 3 and a Phase 1 study evaluating AVXS-101 in spinal muscular atrophy Type 2. AVXS-101 will compete with nusinersen. Novartis, which purchased AveXis in April, has submitted a BLA and MAA. AVXS-101 does not have a PDUFA Date but has orphan drug, breakthrough therapy and fast track priority designations. The other investigational drug for SMA is Catalyst/BioMarin Pharmaceutical’s amifampridine (Firdapse) with a PDUFA Date of Nov 28, 2018. Amifampridine has orphan drug and breakthrough therapy Priority Designations. Amifampridine is approved in the EU. There are nine published studies for amifampridine and one published study for AVXS-101 in our knowledgebase. Click on this link for one month’s access to the Pharmaceutical Pipeline tracker @ $115 with no long-term commitment to review both drugs’ published studies: https://www.prescriberight.com/subscription-offers.html
Novartis announced that in a Phase III trial, alpelisib in combination with fulvestrant was not significantly better than fulvestrant monotherapy in the overall population of men and postmenopausal women with HR+/HER2- advanced breast cancer. In a subset of 341 who harbored PIK3CA mutations, PFS was 11 months with alpelisib and 5.7 months with placebo.
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