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Monthly News Roundup - April 2018

Jynarque is First Approved Drug for Serious Genetic Kidney Disease

The U.S. Food and Drug Administration (FDA) has approved Otsuka’s Jynarque (tolvaptan), a selective vasopressin V2-receptor antagonist, as the first medicine to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD). In this condition, fluid-filled cysts develop in the kidneys leading to kidney failure. Data from Phase III studies showed that Jynarque met the primary endpoints and slowed the decline of kidney function. Due to possible liver injury Jynarque is available only through a restricted distribution program called the Jynarque REMS. Jynarque will be sold at a wholesale acquisition cost of $13,041.10 per 28-day pack.

Crysvita is First Drug Approved for Rare, Inherited Form of Rickets

X-linked hypophosphatemia (XLH) is a rare, inherited form of rickets that leads to low phosphorus levels and impaired bone growth in children; treatment with vitamin D is ineffective. In April, the FDA approved Crysvita (burosumab), a fibroblast growth factor 23 (FGF23) blocking antibody from Ultragenyx Pharmaceuticals. In placebo-controlled studies, 94% of adults receiving Crysvita once a month, and 94% to 100% of children treated with Crysvita every two weeks, achieved normal phosphorus levels. Common side effects in adults included back pain, restless leg syndrome, dizziness, and constipation; in children injection site reactions, vomiting, and fever occurred. Both adults and children experienced headaches and decreased vitamin D levels.

Intravenous Form of Akynzeo Cleared for Chemo-Induced Nausea/Vomiting

Currently, the combined use of antiemetic medicines with different mechanisms is recommended for the prevention of chemotherapy-induced nausea and vomiting (CINV). This month, the FDA approved Helsinn Healthcare’s Akynzeo for Injection (IV) (fosnetupitant and palonosetron), a substance P/neurokinin-1 (NK-1) receptor antagonist and serotonin-3 (5-HT3) receptor antagonist indicated for the prevention of acute and delayed CINV. In studies, bioequivalence was demonstrated between the IV prodrug fosnetupitant and the oral formulation of netupitant, and a safety study revealed no issues with severe allergy or injection site reactions. Helsinn plans to launch Akynzeo IV in May 2018. The oral formulation of Akynzeo was approved in 2014.

FDA Clears Rigel’s Tavalisse for Chronic Immune Thrombocytopenia (ITP)

In immune thrombocytopenia (ITP), the immune system destroys platelets used in blood clotting and healing, leading to excessive bruising, bleeding and fatigue. Tavalisse (fostamatinib) is an oral spleen tyrosine kinase (SYK) inhibitor for the treatment of ITP in patients with insufficient response to a previous treatment. Tavalisse blocks an immune signal that can lead to platelet destruction. Studies included data from 163 ITP patients with a safety database of over 4,600 subjects across other indications. Common side effects were diarrhea, high blood pressure, nausea, dizziness and elevated liver enzymes, among others.

Tagrisso Now First-Line for Lung Cancer with Specific Genetic Markers

The FDA has approved AstraZeneca's Tagrisso (osimertinib), an epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI), for the 1st-line treatment of patients with metastatic EGFR-mutated non-small cell lung cancer (NSCLC), specifically exon 19 deletions or exon 21 L858R mutations. In the Phase III FLAURA trial, Tagrisso 80 mg orally once daily was compared to first-line EGFR-TKI erlotinib (Tarceva) or gefitinib (Iressa) in untreated patients and met the primary endpoint of progression-free survival (PFS) -- 18.9 months (osimertinib ) vs. 10.2 months (standard EGFR-TKI). The most common adverse reactions in studies included diarrhea, rash, dry skin, nail toxicity, mouth sores and swelling (stomatitis), fatigue and decreased appetite.

Posted: April 2018