A light week, until Friday, December 21, then everything happened at once. Of the three drugs with December PDUFA dates, two were approved and one was delayed. The FDA also approved two drugs that did not have PDUFA dates to bring the 2018 total to 59.
The FDA approved prucalopride (Motegrity from Shire) on 12/14/18 for the treatment of adults with chronic idiopathic constipation.
The FDA approved calaspargase pegol (Asparlas from Servier Pharmaceuticals) on 12/20/2018 to treat acute lymphoblastic leukemia as part of a multi-drug regimen.
The FDA approved ravulizumab (Ultomiris from Alexion Pharmaceuticals) on 12/21/2018 for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
The FDA approved tagraxofusp (Elzonris from Stemline Therapeutics) on 12/21/2018 for the treatment of blastic plasmacytoid dendritic cell neoplasms.
The FDA delayed the PDUFA date for solriamfetol by three months to 5/20/2019 to have more time to review draft labeling submitted by Jazz.
Apellis halted enrollment in the DERBY and OAKS Phase III APL-2 trials in October 2018 due to the occurrence noninfectious inflammation. An investigation by Apellis determined the cause to be an impurity or contaminant from a change in the manufacturing process after Phase II trials. A correction has been made and Apellis plans to resume both trials in 2019. The FDA designated APL-2 as an orphan drug.
DBV withdrew its NDA for Viaskin Peanut to correct FDA deficiencies in its manufacturing procedures and quality control.
Nabriva submitted an NDA for oral and IV lefamulin in December 2018.
Serepta complete an NDA submission for golodirsen in December 2018.
Aimmune submitted a BLA for AR101 in December 2018 and plans to file an MAA in 2019.
Menarini licensed pracinostat from the Helsinn Group to promote and distribute the drug. Helsinn will continue to be responsible for development and manufacturing pracinostat.
In a 6-week, 59 patient, Phase II trial, treatment with nabiximols resulted in an improvement of 0.11 points on the Modified Ashworth Scale compared to a decrease of 0.16 with placebo in patients with amyotrophic lateral sclerosis.
In a 16-week, 75 patient, Phase IIa trial, hepatic fat fraction was reduced with pegbelfermin 10 mg daily (-6·8% vs -1·3%) and pegbelfermin 20 mg weekly (-5·2% vs -1·3) compared to placebo in patients with non-alcoholic steatohepatitis.
Chugai announced that in the 18-month, 83 patient, Phase III SAkuraSky trial, 91.5% of patients treated with satralizumab were relapse free at 48 and 96 weeks compared to 59.9% and 53.3% with placebo in patients with neuromyelitis optica spectrum disorder that were receiving immunosuppressive therapy. Chugai announced that in the 18-month, 90 patient, Phase III SAkuraStar trial, treatment with satralizumab increased the time to relapse compared to placebo in patients with neuromyelitis optica spectrum disorder. The FDA designated satralizumab as a breakthrough therapy.
Novartis has the ongoing Phase III STRIVE study evaluating intravenous AVXS-101 in spinal muscular atrophy (SMA) Type 1, 2 & 3 and Phase 1 STRONG study evaluating AVXS-101 in SMA Type 2. AVXS-101 will compete with nusinersen. Novartis bought AveXis in April 2018. The FDA accepted the BLA for AVXS-101 in December 2018. Novartis indicated it may price AVXS-101 at $4-5 million or more for the single dose treatment of SMA, but an analysis by ICER estimated a cost of $2 million would result in an estimated cost of $247,000 per QALY gained and the estimated cost per LY gained of $177,000 in patients with symptomatic Type I SMA.
Clinuvel announced that in a 6-month, 18 patient, Phase trial, afamelanotide administered monthly plus ultraviolet B therapy (NB-UVB) administered twice a week resulted in follicular repigmentation in all body areas, except the feet in Asian patients with vitiligo. Patients received 6 months of afamelanotide and 7 months of NB-UVB (day 196). Pigmentation was maintained through day 280. Three patients dropped out of small Phase II trial due to concerns over darker constitutional skin after drug administration.
AZ announced that 52-week, 2,133 patient Phase III ROCKIES trial, treatment with roxadustat improved hemoglobin in weeks 26-52 compared to placebo in patients with anemia from chronic kidney disease.
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