Exciting news from ASHP Mid-Year, three FDA Approvals, four FDA announcement and six trial and research updates during the week ending December 1, 2018

Exciting news from ASHP Mid-Year. Joanne Stubbings, B.S. Pharm., MHCA, announced the creation of the Section of Specialty Pharmacy Practitioners. It’s the first new section created in twelve years. Three thousand AHSP members have already registered representing a cross section of health system pharmacy. The section’s mission is to advance the cause of health system-based specialty pharmacy practice. Three advisory groups have been created: workforce development, business development, and outcomes and values. 

The FDA approved Loxo Oncology/Bayer’s larotrectinib (Vitrakvi) on 11/26/18 for the treatment of adult and pediatric locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion regardless of site. Larotrectinib is dosed as a 100 mg capsule twice a day in adults and 100 mg/Meter-Squared orally twice daily in children. WAC for a 30-day supply of the drug will be $32,800 for adult dose capsules and start at $11,000 per month for a pediatric liquid. This is the second biomarker guided therapy after the approval of pembrolizumab last May for the treatment of microsatellite instability-high cancer in any location. Bayer and Loxo provide a value-based contract for larotrectinib that will refund payers and patients for patients who do not see a clinical benefit within the first three months of treatment. The companies will also offer patient assistance and reimbursement programs to lower patient costs. Loxo estimates there are 1,500 and 5,000 late-stage cancer patients in the U.S. and a similar number in Europe with the mutation.

The FDA approved amifampridine (Firdapse) on 11/28/18 for the treatment of adults with Lambert-Eaton Myasthenic Syndrome. 

The FDA approved gilteritinib (Xospata) on 11/28/18 for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation.

The FDA has assigned Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib a PDUFA date of 5/25/19. The FDA granted Orphan Drug, Breakthrough Therapy, and Fast Track priority designations to the drug.
The FDA informed CTI that a Phase III myelofibrosis trial will need to be performed before submitting an NDA for pacritinib. CTU plans to start the trial in early 2019. CTI submitted an MAA to the Committee for Medicinal Products for Human Use (CHMP).

The FDA's Endocrinologic and Metabolic Drugs Advisory Committee will review sotagliflozin in mid-January.

The FDA's Reproductive and Urologic Drugs Advisory Committee review romosozumab in mid-January.

In a Phase III trial, patients with obstructive sleep apnea treated with Jazz’s solriamfetol maintained their improvement through 4 weeks in the Maintenance of Wakefulness Test and Epworth Sleepiness Scale at 6 weeks compared to worsening with placebo in patients with excessive sleepiness.  
 
It was a busy news week for Alkermes. 

• In a Phase I, crossover trial, Alkermes’ samidorphan demonstrated abuse potential less than oxycodone and pentazocine and similar to placebo and naltrexone. In another Phase I, crossover trial, samidorphan/buprenorphine 2mg/2mg demonstrated abuse potential similar to placebo, while supratherapeutic doses of 8mg/8mg and 16mg/16mg showed a slight increase compared to placebo, but less than buprenorphine 8 mg or 16 mg alone. 
• In a Phase II trial, olanzapine plus samidorphan had a PANSS score reduction that was equivalent to olanzapine monotherapy, but weight gain was 37% lower with the combination in patients with stable schizophrenia. 
• In a 403 patient, 4-week, phase III trial, olanzapine plus samidorphan demonstrated an improvement in the PANSS score compared to placebo and similar to olanzapine monotherapy in patients with acute exacerbations of schizophrenia. Lastly, Alkermes announced that in the 6-month, 561 patient, Phase III ENLIGHTEN-2 trial, patients treated with olanzapine plus samidorphan had less weight gain that patens treated with olanzapine monotherapy in patients with stable schizophrenia.

Vertex announced interim 4-week data from a 24-week, 380 patient, Phase III trial, where VX-659 in combination with tezacaftor/ivacaftor demonstrated a mean improvement in ppFEV1 of 13% compared to a 1% loss with placebo in cystic fibrosis patients with one F508del mutation and one minimal function mutation. Vertex announced interim 4-week data from a 24-week, 111 patient, Phase III trial, the addition of VX-659 to a combination of tezacaftor/ivacaftor demonstrated a mean improvement in ppFEV1 of 10.2% compared to a 0.3% improvement with placebo in cystic fibrosis patients with two F508del mutations already receiving tezacaftor and ivacaftor.

J&J announced that in a Phase III, rheumatoid arthritis trial, ACR20 was achieved at 1-year by 76.9% of patients treated with sirukumab 50 mg every 4 weeks and 76.9% treated with 100mg every 2 weeks. After two years, 68.3% of patients treated with sirukumab 50 mg every 4 weeks and 74.6% treated with 100mg every 2 weeks maintained ACR20.
In a non-randomized, non-controlled, Phase III safety study, Urovant/Kyorin/Merck’s vibegron improved daily mean micturitions, urgency episodes, urgency incontinence episodes, incontinence episodes and night-time frequency from baseline in patients with overactive bladder. In another Phase III trial, vibegron improved daily mean micturitions, urgency episodes, urgency incontinence episodes, incontinence episodes and night-time frequency compared to placebo in patients with overactive bladder.

In a Phase II trial, 46% of patients treated with Acacia Pharma’s  amisulpride had no emesis or use of rescue medication compared to 59% with a standard triple therapy regimen and 20% with placebo in patients at risk for the delayed phase of chemotherapy-induced nausea and vomiting in chemotherapy-naive patients receiving cisplatin or an anthracycline-cyclophosphamide regimen for breast cancer.

Karyopharm announced interim results from the first 115 or 127 patients in the Phase IIb, SADAL trial, where treatment with selinexor resulted in a 29.6% Overall Response Rate in patients with relapsed or refractory diffuse large B-cell lymphoma.

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