Emerging pharma companies are an important source of product innovation for the pharmaceutical industry, so which companies are making the mark? DCAT Value Chain Insights takes an inside look.

For new molecular entities approved by the US Food and Drug Administration thus far in 2018, more than 60% have originated from small-to-mid-sized innovator pharmaceutical companies, continuing a recent trend. DCAT Value Chain Insights examines key recent approvals.

Emerging pharma muscle

As of the end of July (2018), 26 new molecular entities (NMEs) have been approved by the US Food and Drug Administration’s Center for Drug Evaluation and Research (see Table I at the end of article). Of these 26 NMEs, 16 or 62% were from small-to-mid-sized innovator pharmaceutical companies. Among the companies receiving approval were: Achaogen, Agios Pharmaceuticals, AkaRx, Array BioPharma, BioMarin Pharmaceutical, Fresenius Kabi USA, GW Research, Helsinn Healthcare, Medicines Development for Global Health, Rigel Pharmaceuticals, SIGA Technologies, TaiMed Biologics USA, Ultragenyx Pharmaceutical, US WorldMeds, and Vertex Pharmaceuticals (see Table I at end of article).

One drug of particular note is Epidiolex (plant-derived cannabidiol) by GW Pharmaceuticals, a Cambridge, UK-based pharmaceutical company, is slated for blockbuster status for treating two forms of epilepsy: Lennox-Gastaut syndrome, a form of severe epilepsy that is characterized by multiple types of seizures and intellectual disability, and Dravet syndrome, another severe form of epilepsy. Following a new drug application submission to the FDA in June 2017, GW Pharmaceuticals’ Epidiolex (plant-derived cannabidiol) has the potential to become the first cannabinoid-based anti-epileptic medication. The FDA has granted the drug priority review with a PDUFA review date in June 2018. A marketing-authorization application for the drug in the EU was also submitted in December 2017. Clarivate projects 2022 sales at $1.191 billion.

Of the 26 NME approvals thus far in 2018, 10 or 38% were from the pharmaceutical majors (see Table I at end of article). AbbVie received approval for Orilissa (elagolix sodium), a drug for the management of moderate to severe pain associated with endometriosis. Amgen received approval for Aimovig (erenumab-aooe), a drug to prevent migraine in adults, and AstraZeneca gained FDA approval for Lokelma (sodium zirconium cyclosilicate), a drug to treat hyperkalemia (high potassium levels). Eli Lilly and Company received approval for Olumiant (baricitinib) for treating moderately to severely active rheumatoid arthritis, and GlaxoSmithKline got the FDA nod for Krintafel (tafenoquine), a drug to treat malaria. Gilead Sciences gained an FDA nod with approval for a combination antiviral drug, Biktarvy (bictegravir, embitcitabine, and tenofovir alafenamide) for treating infection in adults who have no antiretroviral treatment history or to replace the current antiretroviral regimen. Johnson & Johnson’s Janssen Pharmaceutical received FDA approval for Erleada (apalutamid) for treating non-metastatic, castration-resistant prostate cancer. Merck & Co. received approval for Ilumya (tildrakizumab) for treating moderate-to-severe plaque psoriasis. Novartis, through its acquisition of Advanced Accelerator Applications in January 2018, added to its oncology portfolio with Lutathera (lutetium Lu 177 dotatate), a drug to treat gastroenteropancreatic neuroendocrine tumors, a type of cancer that affects the pancreas or gastrointestinal tract. Shionogi received approval for Mulpleta (lusutrombopag), a drug to treat thrombocytopenia (low blood platelet count) in adult patients with chronic liver disease.