Update for the Week Ending May 2, 2020

Regulatory Update
 
The FDA approved opicapone (Ongentys, Neurocrine Bioscience) on 4/27/2020, as add-on therapy to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes.
 
The FDA accepted the NDA for terlipressin (Lucassin, Mallinckrodt, Biovie) to treat hepatorenal syndrome type 1 (HRS-1) and set a PDUFA date of 9/12/2020.
 
The EMA accepted the Marketing Authorization Application (MAA) for lonafarnib (Sarasar, Eiger BioPharmaceuticals, Merck) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies.
 
Chimerix began a rolling NDA for brincidofovir as a treatment for smallpox and expects to complete it by mid-2020.
 
Announced Research Updates
 
Roche announced that in the 12-month, 41 patient, Phase III, open-label FIREFISH trial, 29.3% (12/41) of infants treated with risdiplam were able to sit without support for at least five seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) compared to none in a historical control cohort of infants with Type 1 spinal muscular atrophy.
 
Novartis announced that in the 97 patient, Phase II, Geometry mono-1 trial, treatment with capmatinib resulted in an overall response of 40.6% in the pre-treated group and 67.9% in the treatment-naive group in patients with metastatic MET exon 14 skipping non-small cell lung cancer. In 13 patients with brain metastases, 7/13 (54%) had an intracranial response with four patients having a complete resolution of all brain lesions.
 
Published Research Updates
 
In a 4-week, 1,432 patient, open label trial, 70.5% of patients treated with nabiximols had a decrease in at least one spasticity-related symptom in patients with multiple sclerosis drug-resistant spasticity. This observational study was performed in Italy, where the drug is already approved.
 
In a 48-week, 48 patient, phase II trial, treatment with ublituximab resulted in median B cell depletion of > 99%, a decrease in mean T2 lesion volume of 10%, and an annualized relapse rate of 0.07, with 93% of patients with relapsing multiple sclerosis being relapse free.
 
In the 25 patient, Phase II, open-label, PIKTAM trial, treatment with buparlisib plus tamoxifen resulted in a progression-free survival rate at 6-months of 33.3% in patients with hormone receptor-positive, HER2-negative advanced breast cancer previously treated with hormone therapy. The PIKTAM trial was originally designed to enroll 99 patients, but was terminated early due to liver toxicity observed in the BELLE-2 trial.