Emerging Pharma: Who is Rising to the Top?

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.


Leave a Reply

Your email address will not be published. Required fields are marked *

Recent Feature Articles

Teva’s President and CEO Launches New Pivot to Growth Strategy

Teva’s President and CEO, Richard Francis, who assumed the helm of Teva in January, outlined this week the company’s new strategic framework to drive growth. The company’s Pivot to Growth strategy encompasses four main pillars to deliver short- and long-term growth from the company’s commercial portfolio and biosimilars, innovative pipeline, generics business, and focused capital allocation. 

Tackling Drug Shortages and Supply-Chain Vulnerabilities in the European Union

Leaders in the European Union (EU) are addressing ways to mitigate drug shortages and supply-chain vulnerabilities in the EU with 19 EU member states supporting a Critical Medicines Act to reduce the EU’s dependence of non-EU countries for APIs, intermediates and other pharma ingredients. What other actions is the EU taking to mitigate supply-chain vulnerabilities?

CDMOs/CMOs: A Roundup of the Latest Expansions in Biomanufacturing

Several CDMOs/CMOs are proceeding with large-scale investments in new biomanufacturing facilities as biologic drug-substance manufacturing continues to be an active area of investment among CDMOs/CMOs. What companies are expanding production capacity? 

AI & Drug Mfg: Industry Gives Input on FDA’s Discussion Paper

The bio/pharma industry and other stakeholders have provided input on a recent FDA discussion paper on artificial intelligence (AI) in drug manufacturing as the agency considers a regulatory framework and issues to consider when overseeing AI-based drug manufacturing.