The FDA approved teprotumumab (Tepezza, Horizon Therapeutics) on 1/21/2020, for the treatment of thyroid eye disease. Horizon has set WAC for teprotumumab at $14,900 per vial. A full 6-month course of treatment will require 23 vials for a total treatment WAC of $343,000 and an estimated price after discount of $200,000. Teprotumumab was approved 6 weeks early. The original PDUFA date was 3/8/2020.
The FDA approved tazemetostat (Tazverik, Epizyme) on 1/23/20, for the treatment of metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. Tazemetostat is given orally twice a day. Epizyme set WAC at $15,500 for a one-month supply of tazemetostat.
GlaxoSmithKline’s belantamab mafodotin was granted a priority review for the treatment of heavily pre-treated patients with relapsed or refractory multiple myeloma. The drug has a projected summer 2020 PDUFA date.
The FDA designated AstraZeneca’s tremelimumab an Orphan Drug when given in combination with durvalumab for the treatment of hepatocellular carcinoma.
GlaxoSmithKline licensed the M72/AS01E tuberculosis vaccine to the Bill & Melinda Gates Medical Research Institute (MRI). Gates MRI will assume responsibility for development of the vaccine for use in low-income countries with high TB rates.
Announced Research Updates
Roche announced that in the 12-month, 41 patient, Phase III open-label FIREFISH trial, treatment with risdiplam increased the number of infants sitting 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) in patients with Type 1 spinal muscular atrophy.
BeiGene announced interim results from a 360 patient, open-label, Phase III trial, in which progression-free survival was improved in patients treated with tislelizumab plus paclitaxel and carboplatin or tislelizumab plus nab-paclitaxel and carboplatin compared to paclitaxel and carboplatin alone in patients with squamous non-small cell lung cancer.
Ipsen paused two fibrodysplasia ossificans progressiva (FOP) studies when an independent data monitoring committee concluded the current protocol was unlikely to achieve its objective and the protocol and efficacy analysis may need to be modified.
Halozyme announced that in a 494 patient, Phase III trial, treatment with pegvorhyaluronidase alfa plus gemcitabine and nab-paclitaxel did not improve overall survival compared to gemcitabine and nab-paclitaxel (11.2 months vs 11.5 months) alone in patients with metastatic, hyaluronan-high pancreatic ductal adenocarcinoma.
Published Research Updates
In a 50 patient, Phase II trial, veliparib added to cisplatin and gemcitabine did not improve the response rate compared to cisplatin and gemcitabine alone in patients with BRCA/PALB2+ pancreatic ductal adenocarcinomas.
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