The FDA approved voxelotor (Oxbryta, Global Blood Therapeutics) on 11/25/2019 for the treatment of sickle cell disease in patients 12 years and older. The drug had a PDUFA Date of 2/26/2020 and the FDA gave voxelotor Orphan Drug, Fast Track and Breakthrough Therapy Priority Designations.
The FDA granted a priority review for risdiplam (RG7916) targeting spinal muscular atrophy and set a PDUFA date for 5/24/2020. It is the only drug in our knowledge base targeting spinal muscular atrophy.
The FDA assigned pemigatinib a Priority Review for the treatment of locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements and assigned a PDUFA date of 5/30/2020.
CymaBay has terminated clinical trials evaluating seladelpar as a treatment for non-alcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC) and put primary biliary cholangitis (PBC) studies on hold while it investigates atypical liver histological findings. There are eleven other investigational drugs in the Pharmaceutical Pipeline Tracker targeting NASH.
Announced Research Updates
ChemoCentryx announced that in the 52-week, 331 patient, Phase III, ADVOCATE trial, 72.3% of patients treated with avacopan achieved remission [Birmingham Vasculitis Activity Score of zero and no glucocorticoid treatment for anti-neutrophil cytoplasmic antibody-associated (ANCA) vasculitis for at least the preceding four weeks] at 26 weeks compared to 70.1% with corticosteroids. Additionally, 65.7% of avacopan patients remained in remission at 52 weeks compared to 54.9% with glucocorticoids.
Published Research Updates
In a 141 patient, Phase IIb, open-label trial, eryaspase plus either gemcitabine or FOLFOX treated patients had an overall survival (OS) of 6.2 months compared to 4.9 months with standard chemotherapy and progression-free survival (PFS) of 2 months compared to 1.8 months in patients with advanced pancreatic adenocarcinoma with low asparagine synthetase (ASNS) expression. In the overall population OS was 6 months compared to 4.4 months and PFS was 2 months compared to 1.6 months.
AstraZeneca/Lilly’s lanabecestat 104-week, 2,218 patient, Phase II/III, AMARANTH trial and the 78-week, 1,722 patient, Phase III DAYBREAK-ALZ trial were both terminated early after an interim analysis failed to demonstrate an improvement or slowing in decline in the Alzheimer Disease Assessment Scale–cognitive subscale (ADAS-Cog13) compared to placebo in patients with early or mild Alzheimer’s disease.
In the 107-week, Phase III SAkuraSky trial, of 83 patients with neuromyelitis optica spectrum disorder (NMOSD) that were receiving immunosuppressive therapy, 80% of patients treated with satralizumab were relapse free compared to 57% of those given placebo.
In the 681 patient, Phase II/III PALM Trial, mortality was 33.5% after treatment with REGN-EB3, 35.1% after MAb114, 53.1% with remdesivir compared to 49.7% with porgaviximab (ZMapp) in patients infected with the Ebola virus.
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