Ten Items of Interest from over 200 Informational feeds from around the Pharmaceutical Pipeline ecosphere:
1. FDA OKs Tafenoquine (Krintafel) to Prevent Malaria Relapse
GlaxoSmithKline (GSK) and Medicines for Malaria Venture (MMV) announced FDA approval of tafenoquine (Krintafel) to prevent relapse of Plasmodium vivax malaria in people aged 16 years and older who are receiving appropriate antimalarial therapy for acute P vivax infection.
Tafenoquine is the first new agent for P vivax malaria in over 60 years and the only single-dose medicine for this type of relapsing malaria, according to GSK. Tafenoquine was approved under priority review.
2. FDA approves elagolix (Orilissa, AbbVie) treatment for endometriosis
Elagolix is the first oral gonadotropin-releasing hormone antagonist specifically developed for treatment of moderate to severe pain associated with endometriosis. It was approved via priority review and is expected to be available in the US next month.
The drug, to be sold under the name Orilissa, will carry a list price of about $850 per month, or just over $10,100 per year. The price tag falls within the Institute for Clinical and Economic Review's value-based price benchmark range of $8,800 to $12,800, but could still be a strain on the healthcare system in the short-term due to the number of women with endometriosis.
3. Update on VY-AADC for Parkinson’s
Voyager Therapeutics announced likely plans to request approval of VY-AADC as a Parkinson’s gene therapy, based on feedback it received from the FDA and eventual outcomes of a newly opened Phase 2 trial. The company also reported that its Phase 1 trial of VY-AADC showed improvements in patients’ motor function and reduced medication use, along with other benefits, supporting a future biologics license application (BLA).
4. New Analysis of Patisiran Clinical Results
Alnylam presented new analyses of results from APOLLO Phase 3 Study at the 2018 Peripheral Nerve Society Annual Meeting. Patisiran-treated patients showed improvements in overall health status compared to placebo. Indirect analysis demonstrated patisiran’s impact on neuropathy and Quality of Life relative to tafamidis. The Committee for Medicinal Products for Human Use (CHMP) has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. If approved by the European Commission (EC), the medicine will be commercialized under the brand name ONPATTRO™.
5. Merck Released Data on Doravirine, a Potential HIV Treatment
Merck posted 96-week data on the HIV prospect doravirine it hopes will replace other drugs in combination regimens. The update comes with the PDUFA date just three months away.
6. PRIME Designation of Setmelanotide for Genetic Obesity Disorders
Rhythm Pharmaceuticals, Inc, announced that the European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) designation for setmelanotide, the company's first-in-class melanocortin-4 receptor (MC4R) agonist, for treatment of obesity and control of hunger associated with rare deficiency disorders of the MC4R pathway.
7. Orphan Drug Status Recommended for OMS721
The Omeros Corporation announced that the European Medicines Agency's (EMA's) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending orphan drug designation of OMS721 for treatment in hematopoietic stem cell transplantation.
8. Fast Track Designation of APL-2 for Macular Degeneration
Apellis Pharmaceuticals Inc. announced that the FDA has granted Fast Track designation to their APL-2, a novel inhibitor of complement factor C3 as a next generation monotherapy for treatment of geographic atrophy (GA), an advanced form of age-related macular degeneration.
9. Athenex’s Skin Ointment for Precancerous Lesions
Athenex reported that two Phase 3 trials of its skin ointment for precancerous lesions saw 100% clearance of actinic keratosis growths within 60 days, including in the face and scalp.
10. Positive Opinion on Medicine for Complicated Intraabdominal Infections
Tetraphase Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending Xerava (eravacycline) for approval as a treatment for adult patients with complicated intra-abdominal infections (cIAI). The CHMP’s opinion will be reviewed by the European Commission (EC); a final decision is expected within three months. The CHMP opinion is based on data from IGNITE1 and IGNITE4, part of the Phase 3 Investigating Gram-negative Infections Treated with Eravacycline (IGNITE) program.
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