Update for the Week Ending February 29, 2020

Regulatory Update
 
The FDA approved rimegepant (Nurtec ODT, Biohaven) on 2/27/2020 for the acute treatment of migraine with or without aura in adults. Biohaven expects to have rimegepant available in early March. Rimegepant will compete with the triptans, ubrogepant, and lasmiditan. The gepants (ubrogepant and rimegepant) and lasmiditan do not cause vasoconstriction and may be an alternative when patients do not tolerate triptans. Merck abandoned work on two early gepants due to concerns over elevation in transaminase levels with telcagepant, but ubrogepant and rimegepant have not demonstrated liver toxicity. 
 
The FDA approved amisulpride (Barhemsys, Acacia Pharma) on 2/26/2020 for the prevention and treatment of PONV in adult patients.
 
The FDA’s Oncologic Drugs Advisory Committee voted 2-13 against recommending approval of padeliporfin (Tookad, Steba Biotech) for the treatment of adenocarcinoma of the prostate in February 2020.
 
The FDA granted Lysogene/Sarepta’s LYS-SAF302 Fast Track status for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA).
 
Announced Research Updates
 
Vanda announced that in the 8-week, 341 patient, Phase III, EPIONE trial, tradipitant did not improve the Worst Itch Numeric Rating Scale (WI-NRS) score compared to placebo in patients with atopic dermatitis with chronic pruritus.
 
Menlo announced that in a 10-week, 233 patient, Phase II trial, treatment with selopitant did not improve the Worst Itch Numeric Rating Scale score compared to placebo in patients with chronic pruritus of unknown origin. 
 
Published Research Updates
 
ICER reviewed lasmiditan, ubrogepant and rimegepant and found the drugs to be comparable in efficacy, but not as efficacious as triptans. Lasmiditan was found to have higher rates of dizziness and discontinuation. ICER concluded that lasmiditan, ubrogepant and rimegepant would provide a benefit for patients with cardiovascular disease that have a contraindication to triptans, were not helped by triptans or do not tolerate them. ICER estimates that 10-20% more patients have relief from their migraines with the new drugs compared to placebo. Since triptans are available as generics, ICER felt that triptans would provide a greater benefit at a lower cost than lasmiditan, ubrogepant or rimegepant. To reach a threshold of $150,000 per quality-adjusted life year, ICER estimated an annual cost for lasmiditan of $2,800-$3,200 to be cost- effective compared to the annual lasmiditan WAC of $4,610. For ubrogepant and rimegepant, ICER estimated an annual cost of $4,200-$4,600.  The annual WAC for ubrogepant is $4,896. No pricing has been established for rimegepant, since the drug is not approved.
 
In the 4-week, 30 patient, Phase III, cross-over, MMPOWER-2 trial, treatment with elamipretide did not improve distance traveled in a six-minute walking test (6MWT) compared to placebo in patients with primary mitochondrial myopathy (PMM). 
 
In a 12-week, 253 patient, Phase IIb, dose ranging trial, treatment with gefapixant 50 mg reduced the 12-week Awake Objective Cough Frequency by 37% compared to placebo in patients with chronic cough. Treatment with gefapixant 7.5 mg or 20 mg did not differ from placebo.
 
In a 12-week, 287 patient, Phase IIb, dose ranging trial, treatment with fezolinetant reduced the vasomotor symptom (VMS) score by -0.2 to -0.6 point and the VMS frequency by -1.8 to -2.6 events per day compared to placebo in patients with moderate to severe vasomotor symptoms.
 
In a 16-week, 280 patient, Phase Ib trial, lebrikizumab improved the EASI score by 62.3% in patients receiving 125 mg every four weeks, 69.2% with 250 mg every four weeks and 72.1% for patients receiving 250 mg every two weeks compared to a 41.1% improvement with placebo in patients with moderate-to-severe atopic dermatitis.
 
In a 12.5 month, 217 patient, Phase II, open-label trial, treatment with camrelizumab resulted in an overall response in 14.7% of patients with hepatocellular carcinoma.