Tracking New Drug Approvals in 2019

By Patricia Van Arnum - DCAT Editorial Director

July 16, 2019

The US Food and Drug Administration (FDA) has approved 14 new molecular entities (NMEs) thus far in 2019, behind the pace of last year’s record-setting 59 NME approvals. Which drugs have made the mark thus far and are there potential blockbusters in the mix?

New molecular entities: where the industry stands

The FDA has approved 14 new molecular entities thus far in 2019 (see Table I at the end of article). Although NMEs are not approved per a chronological timetable per se, the 14 NME approvals thus far in 2019 is behind the number of NME approvals in 2018. Last year was a record-setting year for NME approvals by the FDA with 59 NME approvals. As of the mid-year mark in 2018, 20 NMEs had been approved.

In the mix between small molecules and biologics, of the 14 NME approvals thus far, nine or 64% were small molecules and five or 36% were biologics (see Table I at the end of article). The five biologics approved thus far are: AbbVie’s Skyrizi (risankizumab-rzaa) for treating moderate-to-severe plaque psoriasis; Amgen’s Evenity (romosozumab-aqqg) for treating osteoporosis; Evolus’ Jeuveau (prabotulinumtoxinA-xvfs) for treating so-called “frown lines”; Roche’s Polivy (polatuzumab vedotin-piiq) for treating relapsed or refractory diffuse large B-cell lymphoma; and Sanofi’s Cablivi (caplacizumab-yhdp) for treating thrombotic thrombocytopenic purpura, a rare blood-clotting disorder.

Big Pharma: Which companies are leading the NME pack?

Among the large pharmaceutical companies, Novartis leads with three NME approvals thus far in 2019. Novartis received FDA approval for Egaten (triclabendazole), a small molecule to treat fascioliasis, a parasitic infestation that mainly affects the liver; Mayzent (siponimod) to treat relapsing forms of multiple sclerosis; and Piqray (alpelisib) for treating certain types of advanced breast cancer (see Table I at the end of article).

Six other of the large pharmaceutical companies—AbbVie, Amgen, Johnson & Johnson (J&J), Pfizer, Roche, and Sanofi—each had one NME approval. As previously mentioned, AbbVie received approval for Skyrizi (risankizumab-rzaa) for treating moderate-to-severe plaque psoriasis, and Amgen received approval for Evenity (romosozumab-aqqg) for treating osteoporosis; Roche received approval for Polivy (polatuzumab vedotin-piiq) for treating relapsed or refractory diffuse large B-cell lymphoma; and Sanofi got the FDA nod for Cablivi (caplacizumab-yhdp) for treating thrombotic thrombocytopenic purpura, a rare blood-clotting disorder. J&J’s Janssen Biotech received approval for Balversa (erdafitinib) for treating locally advanced or metastatic bladder cancer.

Blockbuster watch for 2019 NME approvals thus far

In a recent analysis, Clarivate Analytics’ Cortellis Drugs to Watch 2019 Report identified seven drugs that are set to enter the market in 2019 and achieve blockbuster status (defined as products with sales of $1 billion or more) by 2023. From that report, only one NME approved thus far in 2019 is expected to achieve blockbuster status by 2023: Boehringer Ingelheim’s/AbbVie’s Skyrizi (risankizumab). Cortellis projects 2023 annual sales of $1.74 billion for Skyrizi (risankizumab), a biologic-based drug for treating psoriasis and part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization globally.

The drug, an interleukin-23 (IL-23) inhibitor, was approved by the FDA and the European Medicines Agency in April 2019 for treating moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. Skyrizi will compete with different treatment modalities, such as topicals, light treatments, and systemic medicines, including entrenched biological agents and biosimilar biologicals, according to the Cortellis analysis. Competing non-biological systemic agents include methotrexate and Celgene’s Otezla (apremilast), an oral anti-inflammatory agent. Competing biological systemic agents include the TNF inhibitors, AbbVie’s Humira (adalimumab) and Amgen’s Enbrel (etanercept), which also have biosimilar versions. Other competitors are: Johnson & Johnson’s Stelara, an IL-12/IL-23 inhibitor; the IL-23 inhibitors of Sun Pharmaceutical’s/Almirall’s Ilumya (tildrakizumab) and Johnson & Johnson’s Tremfya (guselkumab); and Novartis’ Cosentyx (secukinumab), an IL-17 inhibitor.

Other NMEs approved thus far in 2019 also show potential. Evolus’ Jeuveau (prabotulinumtoxinA-xvfs) for treating so-called “frown lines” is a potential competitor in certain applications in the aesthetic neurotoxin market to Allergan’s Botox (botulinum toxin), which had 2018 global sales of $3.58 billion.

J&S’s Janssen’s Balversa (erdafitinib) is a precision medicine for treating previously chemo-treated advanced or metastatic bladder cancer patients whose tumors have an FGFR3 or FGFR2 mutation, making it the first targeted therapy approved for that indication and the first FGFR inhibitor on the market. In its fourth-quarter and full-year 2018 earnings call, the company identified erdafitinib as one of its promising NMEs with potential peak sales of $1 billion.

Another key drug is Novartis’ Mayzent (siponimod) for treating relapsing forms of multiple sclerosis (MS) in adults. On its first-quarter 2019 earnings call, the company identified the drug as a potential blockbuster as well. The drug complements the company’s MS drug Gilenya (fingolimod), which had 2018 global sales of $3.3 billion, but which faces potential near-term generic-drug competition pending patent litigation.

Novartis’ Piqray (alpelisib), a drug to certain types of advanced or metastatic breast cancer in postmenopausal women and men, was also identified by Novartis as a potential blockbuster in its first-quarter 2019 results. The drug is specifically indicated for treating hormone receptor-positive, human epidermal growth factor receptor 2-negative, PIK3CA-mutated, advanced or metastatic breast cancer in postmenopausal women and men.

Other drugs noted by analysts with blockbuster potential include Pfizer’s Vyndaqel (tafamidis meglumine) for treating heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis in adults.

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 as of July 3, 2019.
Company Brand name (active ingredient); application type Indication
AbbVie Skyrizi (risankizumab-rzaa); BLA Moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy
AMAG Pharmaceuticals Vyleesi (bremelanotide); NDA Hypoactive sexual desire disorder in premenopausal women
Amgen Evenity (romosozumab-aqqg); BLA Osteoporosis in postmenopausal women at high risk of fracture
Evolus Jeuveau (prabotulinumtoxinA-xvfs); BLA Moderate-to-severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients
Jazz Pharmaceuticals Sunosi (solriamfetol); NDA Excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea
Johnson & Johnson’s Janssen Biotech Balversa (erdafitinib); NDA Locally advanced or metastatic bladder cancer in adults
Karyopharm Therapeutics Xpovio (selinexor); NDA Relapsed or refractory multiple myeloma in adults
Novartis Egaten (triclabendazole); NDA Fascioliasis, a parasitic infestation caused by two species of flatworms or trematodes that mainly affect the liver, sometimes referred to as “liver flukes”
Novartis Mayzent (siponimod); NDA Relapsing forms of multiple sclerosis in adults
Novartis Piqray (alpelisib); NDA Hormone receptor-positive, human epidermal growth factor receptor 2-negative, PIK3CA-mutated, advanced or metastatic breast cancer in postmenopausal women and men
Pfizer Vyndaqel (tafamidis meglumine); NDA Heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis in adults
Roche’s Genentech Polivy (polatuzumab vedotin-piiq); BLA Relapsed or refractory diffuse large B-cell lymphoma in adults
Sage Therapeutics Zulresso (brexanolone); NDA Postpartum depression in adult women
Sanofi Cablivi (caplacizumab-yhdp); BLA Thrombotic thrombocytopenic purpura

Abbvie’s Skyrizi (risankizumab) is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization globally.

Amgen and UCB formed a collaboration and license agreement to research, develop and market antibody products targeting the protein sclerostin in 2004. As part of this agreement, the two companies continue to collaborate on the development of romosozumab for the treatment of osteoporosis.

Jazz Pharmaceuticals has worldwide development, manufacturing, and commercialization rights to Sunosi (solriamfeto), excluding certain jurisdictions in Asia. SK Biopharmaceuticals, the discoverer of the compound, maintains rights in 12 Asian markets, including Korea, China and Japan. .

Pfizer acquired FoldRx in October 2010 and gained Vyndaqel (tafamidis meglumine).

Sanofi acquired Ablynx for $4.8 billion in June 2018 and gained Cablivi (caplacizumab-yhdp).

Source: US Food and Drug Administration and company information