Update for the Week Ending February 1, 2020

Regulatory Update

• The FDA approved the oral peanut-protein immunotherapy, AR101 (Palforzia, Aimmune Therapeutics) on 1/31/2020 for the reduction of allergic reaction incidence and severity in patients aged 4-17 years old with a peanut allergy.
  • Palforzia was approved with a REMS program requiring special training for prescribers and the initial dosing and each dose escalation to be done in a healthcare setting. It is also required that parents or caregivers be counseled on the need to have injectable epinephrine available. 
  • Aimmune set WAC for Palforzia at $890 per month or $10,680 per year. ICER released a review of AR101 and DBV Technologies (DBVT)’s Viaskin Peanut in July 2019. ICER expressed concern regarding the lack of long-term data for safety and effectiveness, desensitization did not eliminate the need to be cautious with peanut exposure, and increase in allergic reaction requiring epinephrine was seen during clinical trials. 
  • The panel also did not find evidence to support AR101 and Viaskin Peanut to have a benefit over avoidance of peanuts or non-commercialized oral immunotherapy. 
  • In a pharmacoeconomic analysis, ICER estimated a benchmark fair-market price of $4,800-7,200 per year for Palforzia and $3,000-$4,500 per year for Viaskin Peanut, which has a PDUFA Date of August 5, 2020.
• The FDA granted a priority review for Lilly, Loxo Oncology’s selpercatinib for the treatment of advanced RET fusion-positive non-small cell lung cancer, RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer and set a PDUFA date in 3Q20.
• The FDA accepted the NDA for Alkermes’ olanzapine/samidorphan for the treatment of schizophrenia and bipolar I disorder and set a PDUFA date in 4Q20.
• PharmaMar has an Expanded Access Program for lurbinectedin to treat U.S. patients with relapsed Small Cell Lung Cancer, who are unable to enter clinical trials and have no alternatives.
• The FDA designated Fulcrum Therapeutics/GlaxoSmithKline’s losmapimod an orphan drug for the treatment of facioscapulohumeral muscular dystrophy.
• The FDA granted Resverlogix’s apabetalone Breakthrough Therapy status in combination with top standard of care, including high-intensity statins, for the secondary prevention of major adverse cardiac events in patients with type 2 diabetes mellitus and recent acute coronary syndrome.
• Bayer submitted an NDA for nifurtimox for the treatment of Chagas disease in January 2020.
• THE EMA accepted the MAA for Gilead Sciences, Kite Pharma’s KTE-X19.
• The EMA accepted the MAA for Seattle Genetics/Cascadian Therapeutics’ tucatinib, combined with trastuzumab and capecitabine, for the treatment of HER2-positive breast cancer.
• CHMP recommended approval of Esperion Therapeutics’ bempedoic acid for the treatment of adults with primary hypercholesterolemia.​
  

Announced Research Updates
 
Sanofi announced that in a 52-week, 36 patient, Phase II/III trial, treatment with olipudase resulted in a relative improvement of 22% in the predicted diffusing capacity of carbon monoxide (DLco) and a 39.5% decrease in spleen size compared to a 3% DLco improvement and a 0.5% decrease in spleen size with placebo in adult patients with acid sphingomyelinase deficiency (Niemann-Pick Disease). However, treatment with olipudase did not improve the Splenomegaly Related Score compared to placebo. Sanofi announced that in a 52-week, 20 patient, open-label, Phase II trial, treatment with olipudase resulted in a relative DLco improvement of 33% and a 49% decrease in spleen size in patients < 18 years old with acid sphingomyelinase deficiency.
 
The NIH announced that it halted a trial after interim data from 5,383 patients enrolled in a 5,407 HIV-negative patient, Phase IIb/III trial, showed that 129/2,694 patients who received the Sanofi Pasteur vaccine with the GSK immunity-boosting adjuvant became infected with HIV compared to 123/2,689 patients that received placebo.
 
Published Research Updates
 
In a 12-week, 111 patient, Phase IIb trial, several dosing regimens of tirzepatide lowered HbA1c more than placebo in patients with type 2 diabetes.
 
In a 13-week, 42 patient, Phase II, open-label trial, treatment with bermekimab resulted in 61% of patients naive to anti-TNF treatment and 63% of patients that failed anti-TNF treatment achieving Hidradenitis Suppurativa Clinical Response (HiSCR), which was defined as a greater than or equal to 50% reduction in inflammatory lesion count and no increase in abscesses or draining fistulas.
 
In an 8-week, 204 patient, Phase II trial, 33.3% of patients treated with serlopitant achieved a 4 point or > worst-itch numeric rating scale (WI-NRS) score compared to  21.1% with placebo in patients with pruritus associated with psoriasis.