First half 2019 New Drug Approvals

During the first half of 2019 the FDA approved 13 investigational drugs for release to the marketplace.

Bremelanotide (Vyleesi) The FDA approved AMAG Pharmaceuticals bremelanotide on 6/21/2019 for the treatment of premenopausal women with acquired, generalized hypoactive sexual desire disorder.
Press Release

Polatuzumab vedotin-piiq (Polivy) The FDA approved polatuzumab vedotin on 6/10/2019 in combination with bendamustine and rituximab for the treatment of adults with resistant diffuse large B-cell lymphoma.
Press Release

Alpelisib (Piqray) The FDA approved alpelisib (Piqray, Novartis) on 5/24/2019 to be used in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer following tumor advancement after endocrine-based regimens. The companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, was also approved to detect the PIK3CA mutation in a tissue and/or a liquid biopsy.
Press Release
Drug Trials Snapshot

Tafamidis meglumine (Vyndaqel) The FDA approved tafamidis meglumine (Vyndaqel) and tafamidis (Vyndamax) early on 5/6/2019 for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. The recommended dosage for tafamidis meglumine is four 20 mg capsules once daily (80 mg total dose). The recommended dose for tafamidis is one 61 mg capsule taken once-daily. The annual WAC for both drugs is $225,000. 
Press Release 
Drug Trials Snapshot

Risankizumab-rzaa (Skyrizi) The FDA approved risankizumab on 4/23/2019 for the treatment of plaque psoriasis. Risankizumab is dosed every 12 weeks after an initial 2 injections. The drug will compete with ixekizumab, tildrakizumab, guselkumab, and secukinumab. ICER has estimated an annual price of $28,800–$42,100 in order for risankizumab to achieve a long-term cost-effectiveness benchmark of $100,000– $150,000 per QALY. ICER suggested that step therapy approaches may not be appropriate for psoriasis. In an analysis of current data ICER found that risankizumab may be more effective than TNF-blockers, but this is based on grey literature, since risankizumab studies have not been published. AbbVie announced the annual WAC for maintenance dosing of risankizumab is $59,000.
Drug Trials Snapshot

Erdafitinib (Balversa) The FDA approved erdafitinib on 4/12/2019 for the treatment locally advanced or metastatic bladder cancer with fibroblast growth factor receptor (FGFR)3 or FGFR2 mutations who have progressed on platinum-containing chemotherapy. A companion lab test was also approved to identify patients with these mutations. WAC for erdafitinib will be $10,080 to $22,680 for a 28-day supply, depending on dose for an annual cost of $131,040 to $294,840.
Press Release
Drug Trials Snapshot

Romosozumab-aqqg (Evenity) The FDA approved romosozumab on 4/9/2019 for the treatment of osteoporosis in postmenopausal women at high risk for fracture. The drug has a Boxed Warning regarding the increased risk of myocardial infarction, stroke and cardiovascular death. Amgen set WAC for romosozumab at $1,825 a month for an annual cost of $21,900. Romosozumab will likely join abaloparatide and teriparatide as a specialty drug for the treatment of osteoporosis. WAC for abaloparatide is $22,000, while the Federal Supply Schedule price is $11,800. For teriparatide, WAC is $36,000, and the Federal Supply Schedule price is $26,000. ICER estimated the cost per quality adjusted life year gained over the bisphosphonate zoledronic acid as $334,000 for abaloparatide and $942,000 for teriparatide. Due to the delay in FDA review, ICER did not include rombosozumab in its review.
Press Release
Drug Trials Snapshot

Siponimod (Mayzent) The FDA approved siponimod (Mayzent, Novartis) on 3/26/2019 to treat adults with relapsing multiple sclerosis. In a draft review ICER felt that compared to best supportive care siponimod reduced the risk of disability progression and decreased inflammation. ICER analysis found that siponimod lowered the risk of relapse by 46% and had an annualized relapse rate of 0.07/year compared to 0.16/year with placebo. Due to the lack of comparative trials with other disease modifying therapies and the difference in patient populations and outcome assessments with current trials, ICER analysts did not feel enough data existed to compare siponimod to those therapies. ICER estimated a quality-adjusted life year (QALY) of $826,000 for use of siponimod over best supportive care in the treatment of secondary progressive multiple sclerosis, assuming the same price as ocrelizumab. The cost per QALY dropped to $396,000 in patients with relapses within two years of the initiation of treatment. ICER reported an annual acquisition price for ocrelizumab of $64,308 after discounts. Novartis has announced an annual WAC of $88,000 for  siponimod. There have been no estimates or reports of potential discounts for siponimod, during the week the drug was approved. ICER will accept comments on the draft report until April 10 and then release a final report on May 2 with a discussion of the report set for May 23.
Press Release
Drug Trials Snapshot

Solriamfetol (Sunosi) The FDA approved solriamfetol on 3/20/2019 to improve wakefulness in adults with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea. 
Drug Trials Snapshot

Brexanolone (Zulresso) The FDA approved brexanolone on 3/19/2019 for the treatment of postpartum depression. 
Press Release
Drug Trials Snapshot

Triclabendazole (Egaten) The FDA approved triclabendazole (Egaten) on 2/13/2019 for the treatment of fascioliasis, a parasitic infestation caused by two species of flatworms or trematodes that mainly the affect the liver, sometimes referred to as “liver flukes”. Prior to approval, the drug has been available through the CDC.
Drug Trials Snapshot

Caplacizumab-yhdp (Cablivi) The FDA approved caplacizumab (Cablivi by Sanofi) on 2/6/2019 for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). 
Press Release
Drug Trials Snapshot

PrabotulinumtoxinA-xvfs (Jeuveau) The FDA approved PrabotulinumtoxinA (Jeuvea from Evolus) on 2/1/2019 for the treatment of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients.
Press Release