Emerging Pharma: Who is Rising to the Top?By
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.
|Table I: Approvals of New Molecular Entities, New Drug Applications (NDAs) and Original Biologics License Applications (BLAs) by the US Food and Drug Administration’s Center for Drug Evaluation and Research as of July 31, 2018.|
|Company||Brand name (active ingredient); application type||Indication|
|AbbVie||Orilissa (elagolix sodium); NDA||Moderate to severe pain associated with endometriosis|
|Achaogen||Zemdri (plazomicin); NDA||Adults with complicated urinary tract infections|
|Advanced Accelerator Applications (Novartis company)||Lutathera (lutetium Lu 177 dotatate); NDA||Gastroenteropancreatic neuroendocrine tumors, a type of cancer that affects the pancreas or gastrointestinal tract|
|Agios Pharmaceuticals||Tibsovo (ivosidenib); NDA||Adult patients with relapsed or refractory acute myeloid leukemia who have a specific genetic mutation|
|AkaRx||Doptelet (avatrombopag); NDA||Low blood platelet count (thrombocytopenia) in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure|
|Amgen||Aimovig (erenumab-aooe); BLA||Preventive treatment of migraine in adults|
|Array BioPharma||Braftovi (encorafenib); NDA||Unresectable or metastatic melanoma|
|Array BioPharma||Mektovi (binimetinib); NDA||Unresectable or metastatic melanoma|
|AstraZeneca||Lokelma (sodium zirconium cyclosilicate); NDA||Hyperkalemia (high potassium levels)|
|BioMarin Pharmaceutical||Palynziq (pegvaliase-pqpz); BLA||Phenylketonuria (inherited disorder that increases the levels of phenylalanine in the blood)|
|Eli Lilly and Company||Olumiant (baricitinib); NDA||Moderately to severely active rheumatoid arthritis|
|Fresenius Kabi USA||Omegaven (fish oil triglycerides); NDA||A source of calories and fatty acids in pediatric patients with parenteral nutrition-associated cholestasis|
|Gilead Sciences||Biktarvy (bictegravir, embitcitabine, and tenofovir alafenamide); NDA||Infection in adults who have no antiretroviral treatment history or to replace the current antiretroviral regimen|
|GlaxoSmithKline||Krintafel (tafenoquine) NDA||Malaria|
|GW Research||Epidiolex (cannabidiol); NDA||Seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome|
|Helsinn Healthcare||Akynzeo (fosnetupitant and palonosetron); NDA||To prevent acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy|
|Janssen Pharmaceutical (Johnson & Johnson)||Erleada (apalutamid); NDA||Non-metastatic, castration-resistant prostate cancer|
|Medicines Development for Global Health||Moxidectin (moxidectin); NDA||Onchocerciasis due to Onchocerca volvulus in patients aged 12 years and older|
|Merck & Co.||Ilumya (tildrakizumab); BLA||Adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy|
|Rigel Pharmaceuticals||Tavalisse (fostamatinib); NDA||Thrombocytopenia (low blood platelet count) in adult patients with persistent or chronic immune thrombocytopenia|
|SIGA Technologies||Tpoxx (tecovirimat); NDA||Smallpox|
|Shionogi||Mulpleta (lusutrombopag); NDA||Thrombocytopenia (low blood platelet count) in adult patients with chronic liver disease who are scheduled to undergo a procedure|
|TaiMed Biologics USA||Trogarzo (ibalizumab-uiyk); BLA||HIV|
|Ultragenyx Pharmaceutical||Crysvita (burosumab-twza); BLA||To treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets|
|US WorldMeds||Lucemyra (lofexidine); NDA||Non-opioid treatment for management of opioid withdrawal symptoms in adults|
|Vertex Pharmaceuticals||Symdeko (tezacaftor and ivacaftor); NDA||Cystic fibrosis in patients age 12 years and older|
Novartis acquired Advanced Accelerator Applications in January 2018.
In August 2015, Amgen entered into a global collaboration with Novartis to jointly develop and commercialize treatments in the fields of migraine and Alzheimer’s disease. The collaboration focuses on investigational Amgen drugs in the migraine field, including Aimovig. In April 2017, the collaboration was expanded to include co-commercialization of Aimovig in the US. For the migraine program, Amgen retains exclusive rights in Japan, and Novartis has exclusive rights in Europe, Canada, and rest of world
Array BioPharma has exclusive rights to Braftovi (encorafenib) and Mektovi (binimetinib) in the US and Canada. Array has granted Ono Pharmaceutical exclusive rights to commercialize both products in Japan and South Korea and Pierre Fabre exclusive rights to commercialize both products in all other countries, including in Europe, Asia, and Latin America.
Source: US Food and Drug Administration’s Center for Drug Evaluation and Research and company information.