Update for the week ending July 27, 2019

Regulatory updates
 
The FDA approved ferric maltol (Feraccru/Accrufer, Shield Therapeutics) on 7/26/2019 for the treatment of iron deficiency in adults.
 
The FDA assigned a priority review for tazemetostat for the treatment of metastatic or locally advanced epithelioid sarcoma and assigned a PDUFA date of 1/23/2020.
 
The FDA accepted the BLA for Sanofi’s isatuximab for treatment of multiple myeloma and assigned a PDUFA date of 4/30/2020.
 
The FDA canceled an advisory committee meeting to review lumateperone as a treatment for schizophrenia due to new information provided for the NDA. No change has been made for the September 2019 PDUFA date.
 
Vertex filed an NDA in July 2019 for VX-445 (elexacaftor, tezacaftor and ivacaftor) for the treatment of cystic fibrosis in patients with one F508del mutation and one minimal function mutation or two F508del mutations.
 
The FDA designated La Jolla’s artesunate (LJPC-0118) an orphan drug for the treatment of malaria.
 
Orphazyme plans to file an NDA for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC) in the first half of 2020. Orphazyme plans to introduce an Early Access Program for NPC in the fall of 2019.
 
The FDA delayed a decision on NKTR-181 to allow additional time to review preclinical data from two additional studies. In July 2019, the FDA put review of NKTR-181 on hold and canceled an advisory committee review of the drug. Some analysts expect a 1-year delay in a review.
 
Announced Research Results
 
Myovant announced that in the 24-week, 382 patient, Phase III, LIBERTY 2 trial, 71.2% of patients treated with relugolix, estradiol and norethindrone acetate achieved a menstrual blood loss volume of less than 80 mL and a 50 percent or greater reduction from baseline in menstrual blood loss volume during the last 35 days of the 24-week treatment period compared to 14.7% with placebo in patients with uterine fibroids and heavy menstrual bleeding.
 
Marinus announced that in Part 2 of the 34-day, 33 patient, Phase II MAGNOLIA trial, treatment with a 6-hour infusion of ganaxolone followed by daily oral administration of the drug did not improve the HAM-D17 score compared to placebo in women with postpartum depression. Marinus announced that in the 28-day, 68 patient, Phase II, open-label, Amaryllis trial, where treatment with oral ganaxolone 675 mg decreased the HAM-D17 score by 0.8, 9.8, and 12.2 at 24 hours, 14 days and 28 days of treatment while increasing the dose to 1,125 mg after two days deceased the HAM-D17 score by 2.7, 9.3 and 14.5 at 24 hours, 14 days and 28 days of treatment in women with postpartum depression.
 
Published Research Results
 
In a 26-week, 256 patient, Phase III trial, roxadustat was non-inferior to epoetin in increasing hemoglobin (0.7g/dL vs 0.5 g/dL) in Chinese dialysis patients previously maintained with epoetin. In a 9-week, 154 patient, Phase III trial, roxadustat increased hemoglobin 1.9 g/dL compared to a decrease of 0.4 g/dL with placebo in Chinese patients with chronic kidney disease that were not receiving dialysis.
 
In the 24-week, 448 patient, Phase III, FINCH 2 trial, 66% of patients treated with filgotinib 200 mg and 57.5% treated with 100 mg achieved AR20 (compared to 31.1% with placebo in patients with with moderate-to-severe rheumatoid arthritis.
 
In a 12-week, 507 patient, Phase III trial, 57.7% of patients treated with peficitinib 100mg achieved AR20, 74.5% with 150mg and 30.7% with placebo in patients in rheumatoid arthritis patients with an inadequate response to at least one conventional DMARD. In a 12-week, 519 patient, Phase III trial, 58.6% of patients treated with peficitinib 100mg achieved AR20, 64.4% with 150mg and 21.8% with placebo in Japanese rheumatoid arthritis patients with an inadequate response to methotrexate.