Update for August 11, 2020

Regulatory Update
 
The FDA approved belantamab mafodotin (Blenrep, GlacoSmithKline), on 8/6/2020 for the treatment of heavily treated relapsed or refractory multiple myeloma. Belantamab mafodotin was approved with a REMS program that requires monitoring and physician and patient education about the ocular risks associated with treatment. In the pooled safety population, ocular adverse reactions occurred in 77% of the 218 patients. The ocular adverse events seen most often include keratopathy (76%), changes in visual acuity (55%), blurred vision (27%), and dry eye (19%). GSK has set WAC at $8,277 per vial for an estimated monthly cost of $23,900.
 
The FDA approved risdiplam (Evrysdi, Genentech), on 8/7/2020, for treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Risdiplam will be available in late August through Accredo Health Group, an Express Scripts specialty pharmacy for direct delivery to patients’ homes. Risdiplam will compete with nusinersen (Spinraza, Biogen), which was approved in 2016. Risdiplam has weight and age based dosing until a child weighs 44 pounds. For patients that weigh 44 pounds or more, the dose is standardized to 5 mg per day. At 5 mg per day, Genentech has priced risdiplam at $340,000 per year. For an infant weighing 15 pounds, the annual cost would be less than $100,000. Both risdiplam and nusinersen increase production of survival motor neuron (SMN) protein. However, nusinersen is administered by intrathecal injection under anesthesia, while risdiplam is an oral liquid. WAC for nusinersen is $750,000 during the first year and $375,000 for each subsequent year.  Onasemnogene abeparvovec (Zolgensma, Novartis) is a single-dose gene therapy for SMA, but the drug has a price of over $2 million and a Black Box Warning regarding liver toxicity.
 
The FDA approved nifurtimox (Lampit, Bayer), on 8/7/2021, for the treatment of Chagas disease (American Trypanosomiasis) caused by Trypanosoma cruzi (T. cruzi) in pediatric patients. The two drugs approved to treat Chagas disease are nifurtimox and benznidazole. Benznidazole was approved in 2017. Bayer plans to offer a program to reduce co-pays to $0.
 
The FDA approved oliceridine (Olinvyk, Trevena), on 8/7/2021 for short-term intravenous use in hospitals or other controlled clinical settings for the management of moderate to severe acute pain in adults. Oliceridine is an opioid and was approved with the opioid boxed warning about addiction, abuse and misuse; life-threatening respiratory depression; neonatal opioid withdrawal syndrome; and risks from concomitant use with benzodiazepines or other central nervous system depressants. Oliceridine is administered intravenously and has a maximum recommended daily dose limit of 27 milligrams.
 
The FDA rejected the Viaskin Peanut desensitizing patch due to concerns over patch adhesion and how it affects efficacy. The FDA requested additional manufacturing data, a redesign of the patch adhesion and a new trial to demonstrate efficacy with the new patch adhesion.
 
The EMA conditionally approved MYR Pharmaceuticals’ bulevirtide for the treatment of chronic hepatitis D with compensated liver disease.
 
Announced Research Updates

Elios Therapeutics announced that in a 3-year, 144 patient, Phase IIb trial, treatment with TLPLDC resulted in disease free survival (DFS) in 51.8% of patients and overall survival (OS) in 92.9% of patients compared to a DFS of 27.1% and OS of 70.3% with placebo in patients with Stage III or Stage IV melanoma at high risk of recurrence following complete surgical resection.
Genentech announced that in the 10-week, 358 patient, Phase III, HIBISCUS I trial, treatment with etrolizumab induced remission in more patients than placebo in patients with moderately to severely active ulcerative colitis who have not been previously treated with anti-TNF agents. However, in the 10-week, 358 patient, Phase III, HIBISCUS II trial, treatment with etrolizumab was no different than placebo in inducing remission in patients with moderately to severely active ulcerative colitis who have not been previously treated with anti-TNF agents. Genentech announced that in the 14-week, 609 patient, Phase III, HICKORY trial, more patients treated with etrolizumab had a successful induction compared to placebo, but etrolizumab was no better than placebo in sustaining remission in patients with moderately to severely active ulcerative colitis who have been previously treated with anti-TNF agents. Genentech announced that in the 62-week, 359 patient, Phase III, LAUREL trial, etrolizumab was no better than placebo in sustaining remission in patients with moderately to severely active ulcerative colitis who have not been previously treated with anti-TNF agents.
 
Published Research Updates
 
41 heavily pretreated patients with relapsed or refractory Hodgkin’s lymphoma were enrolled in two Phase I/II trials and received CD 30 CAR-T cell therapy. One-year progression-free survival was 36% and overall survival was 94%. The overall response rate in 32 patients with active disease who received fludarabine-based lymphodepletion was 72%.
 
In a 12-week, 352 patient, Phase IIb, dose ranging trial, treatment with fezolinetant reduced the Menopause-Specific Quality of Life questionnaire VMS scores by 2.9 to 4.4 compared to a 2.3 point decrease with placebo, reduced the Hot Flash-Related Daily Interference Scale by 3.3 to 4.3 points compared to 2.9 point decrease with placebo and reduced Greene Climacteric Scale-VMS domain scores by 2.1 to 3.3 points compared to a 2.1 point decrease with placebo in patients with moderate to severe vasomotor symptoms.