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Monthly News Roundup - June 2018

First Cannabis-Based Drug Epidiolex Approved for Childhood Seizures

In June, the U.S. Food and Drug Administration (FDA) approved GW Pharmaceuticals' Epidiolex (cannabidiol) [CBD], an oral solution for patients two years and older to treat seizures linked with two rare and severe forms of epilepsy -- Lennox-Gastaut syndrome and Dravet syndrome. Epidiolex is the first FDA-approved drug from a marijuana-based substance. Unlike tetrahydrocannabinol (THC), CBD does not cause intoxication or a 'high'. In pivotal studies Epidiolex was shown to be effective in reducing the frequency of seizures when compared with placebo. Common side effects include sleepiness, elevated liver enzymes, and decreased appetite, among others.

Combined Braftovi + Mektovi Cleared for Advanced Melanoma

In the ongoing fight against metastatic melanoma, the FDA has now given the go-ahead to Braftovi (encorafenib) and Mektovi (binimetinib) from Array BioPharma. The oral BRAF/MEK inhibitor combination therapy is used for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation. In the COLUMBUS trial, the median progression-free survival was 14.9 months for patients receiving the Braftovi and Mektovi combination, compared to 7.3 months for vemurafenib (Zelboraf) monotherapy. Common side effects include fatigue, nausea, diarrhea, vomiting, stomach pain, and joint pain.

Zemdri: The Latest Aminoglycoside for Urinary Tract Infections

The FDA has cleared intravenous Zemdri (plazomicin) for adults with complicated urinary tract infections (cUTI), including pyelonephritis (kidney infection), caused by certain Enterobacteriaceae in patients with few options. Zemdri is an aminoglycoside antibacterial. In the phase 3 EPIC cUTI trial, Zemdri was non-inferior to (not worse than) treatment with meropenem for the two primary endpoints. Clinical cure and microbiological eradication at day 5 was 88% for Zemdri compared to 91.4% for meropenem. Common study side effects included decreased kidney function, diarrhea, and blood pressure changes, among others.

Nocdurna is First Under-the-Tongue Tablet for Frequent Nighttime Urination

This month, the FDA approved Ferring's Nocdurna (desmopressin acetate), a vasopressin analog and the first sublingual (under-the-tongue) tablet for the treatment of nocturia due to nocturnal polyuria in adults who wake up at least two times per night to urinate. The mean baseline voids were 2.9 for women and 3.0 for men. Clinical trials demonstrated an average reduction of nocturnal voids by 52% in women (n=118) and 43% in men (n=102) relative to mean baseline (reduction of 1.5 and 1.3 voids, respectively). Commonly reported side effects included dry mouth, decreased blood sodium, and dizziness.

Olumiant Gains Approval for Moderate-to-Severe Rheumatoid Arthritis

Roughly two-thirds of rheumatoid arthritis (RA) patients will not reach remission with their first tumor necrosis factor (TNF) inhibitor therapy. Eli Lilly and Incyte have announced the approval of Olumiant (baricitinib), a once-daily oral medicine for adults with moderate-to-severe RA and an inadequate response to TNF inhibitors. Like Xeljanz (tofacitinib), Olumiant is a Janus kinase (JAK) inhibitor. Olumiant may be used alone or in combination with methotrexate or other non-biologic DMARDs. In studies, Olumiant had a significant response rate compared to placebo at Week 12 (49% versus 27%, respectively). According to Lilly, the price of Olumiant will be 60% less than the leading TNF inhibitor.

FDA Approves Fulphila, the First Biosimilar to Neulasta

In June, a new biosimilar -- Fulphila (pegfilgrastim-jmbd) from Mylan and Biocon -- was FDA-approved. Fulphila is a biosimilar to Neulasta (pegfilgrastim) and is used to reduce the duration of febrile neutropenia (fever or other signs of infection with a low white blood cell count) in patients treated with chemotherapy for certain types of cancer. As with other biosimilars, the approval for Fulphila was based on data which showed no clinically meaningful differences between the two products in terms of safety, purity and potency.

Posted: June 2018

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