US Product Launches To Plan For In 2020
Executive Summary
A sizeable group of key late-stage products have the potential to reach the US market in 2020, according to a recent report from Informa’s Biomedtracker. Pharmaceutical and biotech executives will have to be on their A-game to integrate the new wave of products into patient-centric and market access-friendly commercial strategies.
Informa’s drug development database Biomedtracker believes that more than 50 candidate medicines, across 15 broad therapeutic categories and involving more than 60 companies, may reach the US market over the next 12 to 18 months, according to its new report.
The products include MacroGenic’s margetuximab, Genfit’s elafibranor, Allergan’s ubrogepant, Merck & Co’s ebola vaccine and GlaxoSmithKline’s antibody-drug conjugate GSK2857916.
The productivity of the pharmaceutical and biotech industries is evident from the report, Key Potential Drug Launches in 2020, which highlights some of the diseases and conditions that could benefit from new therapies during the year, including liver fibrosis, sickle cell anemia, prostate cancer, multiple myeloma, ebola and dengue fever.
The report builds on the quarterly reports already issued by the pharma pipeline analysts and may give clues to what may be changes to standards of care in the years to come, the market opportunity for new products, and how new drug classes may improve patient outcomes. (Also see "Key Drug Launches To Plan For In 2019" - Scrip, 3 Sep, 2018.)
PARP Inhibitors For Prostate Cancer
Of the four PARP inhibitors currently in Phase III studies for prostate cancer, GlaxoSmithKline PLC’s Zejula (niraparib) and Clovis Oncology Inc.’s Rubraca (rucaparib) appear likely to gain the new indication first, the report notes.
Rubraca’s Phase III TRITON3 study is still ongoing but top-line results were positive and Clovis may file for accelerated approval in the near future. Likewise, Zejula’s Phase II GALAHAD study has seen positive top-line data, and partner Janssen Pharmaceutical Cos. could file for approval this year. (Also see "J&J Plans 10 Potential Blockbuster Filings By 2023 " - Scrip, 15 May, 2019.)
PARP inhibitors are likely to be of benefit in castration-resistant prostate cancer patients with BRCA1/2 mutations who have few treatment options after failing therapy on next-generation androgen receptor targeted therapies or chemotherapy, so the commercial potential of the newer agents could be high, the report remarks.
HER2-Positive Breast Cancer
MacroGenics Inc.’s Fc domain-engineered Mab, margetuximab, has been shown to improve progression-free survival (PFS) in top-line data from the Phase III SOPHIA study, and the company expects to file for US approval by the end of 2019.
There are currently no approved medicines for metastatic HER2-positive breast cancer who have received Herceptin (trastuzumab), Perjeta (pertuzumab) and Kadcyla (trastuzumab emtansine), and margetuximab could become a valuable treatment option for third- or fourth-line patients, according to the Biomedtracker report (see sidebar).
Sickle Cell Anemia, Beta-Thalassemia
Starting in 2020, a new wave of products for sickle cell anemia and transfusion-dependent beta-thalassemia could reach the US market, the report notes. These newer agents include Global Blood Therapeutics Inc.’s voxelotor, which increases hemoglobin’s affinity for oxygen, thereby reducing the sickling of erythrocytes.
Others include Novartis AG’s crizanlizumab and Pfizer Inc.’s rivipansel, which are selectin inhibitors currently being evaluated for reducing the rate or extent of vaso-occlusive crises in sickle cell. Gene therapy companies are also making progress in hematology, with bluebird bio Inc.’s Zynteglo possibly being approved for transfusion-dependent beta-thalassemia in 2020. The ligand trap, Acceleron Pharma Inc.’s luspatercept, is likely to be approved for marketing in 2020, the report says.
Already approved in China, the potential anemia therapy, roxadustat, could gain additional approvals in 2020 for three companies involved in its global development, AstraZeneca PLC, FibroGen Inc. and Astellas Pharma Inc..
Merck’s Ebola Vaccine
Merck & Co. Inc.’s ebola vaccine, V920, which is administered in a single-dose schedule and addresses a major unmet medical need, particularly in West Africa, has been submitted for approval in the US and Europe, and the company hopes it will gain prequalification status from the WHO, allowing the vaccine to be bought and distributed by healthcare bodies such as UNICEF and GAVI.
Available data from its use in front-line healthcare workers suggests V920 is effective, although the presence of medical teams in the locality where the candidate vaccine was administered may have induced behavioral changes in the population. (Also see "WHO To Facilitate Rapid Roll Out Of Merck's Ebola Vaccine In Africa" - Pink Sheet, 28 May, 2019.)
Takeda’s Dengue Vaccine
Dengue fever appears to be spreading around the world, and Takeda Pharmaceutical Co. Ltd. is developing a new tetravalent live-attenuated vaccine, TAK-003, to prevent the viral infection, which could gain approval in the US and in selected dengue-endemic Latin American and Asia-Pacific markets starting in late 2020.
TAK-003 is likely to have a more convenient dosing schedule than Sanofi's marketed vaccine, and a better efficacy in seronegative children, the report suggests. The candidate vaccine has the potential to have superior efficacy and safety in young children, and initial studies in children have not raised any safety concerns. (Also see "Takeda Sees Strong Fundamentals Despite Post-Shire Loss Outlook" - Scrip, 14 May, 2019.)
GSK And bluebird bio In Multiple Myeloma
GlaxoSmithKline’s antibody-drug conjugate, GSK2857916 is directed toward the B-cell maturation antigen, BCMA, expressed on the cells of a high proportion of multiple myeloma patients, a novel mode of action, and promising results have been reported so far in initial clinical studies. These could become the basis of initial filings (see sidebar).
bluebird bio Inc.’s idecabtagene vicluecel (bb2121) is a CAR-T therapy targeting BCMA, that has also shown promise in initial studies in multiple myeloma patients, and top-line results from a registration-enabling Phase II KarMMa trial are expected in the second half of 2019. Bluebird is collaborating with Celgene Corp., which is currently merging with Bristol-Myers Squibb Co., on the development of bb2121. The Celgene/BMS transaction is expected to close in the third quarter of 2019. (Also see "Bluebird Lays Some New Eggs, But They'll Take Time To Hatch" - Scrip, 16 May, 2019.)
ViiV’s Long-Acting Anti-HIV Injectable
The first long-acting injectable (LAI) to enter the HIV market is expected to be ViiV Healthcare /Johnson & Johnson’s cabotegravir/rilpivirine. The product could become a blockbuster if patients prefer it to once-daily oral tablets, Biomedtracker points out.
ViiV filed for US approval in April 2019 and an EU filing is expected shortly, based on positive data from the FLAIR and ATLAS studies. (Also see "ViiV/Janssen's Long-Acting Anti-HIV Injections Impress" - Scrip, 16 Aug, 2018.)
Allergan’s Oral Migraine therapy
Allergan PLC’s ubrogepant is in the driving seat to become the first oral, calcitonin gene-related peptide (CGRP) receptor antagonist approved for the acute treatment of migraine in the US, with a PDUFA date of December 2019.
The approval of ubrogepant is expected to be closely followed by Biohaven Pharmaceutical Holding Co. Ltd.’s oral CGRP antagonist, remegepant, although based on pivotal clinical trials, ubrogepant appears slightly superior to rimegepant with regards to efficacy, the report says. (Also see "Biohaven Pays $105m For Turbo Boost In Migraine Drug Review Race" - Scrip, 20 Mar, 2019.)
Intercept & Genfit’s NASH Therapies
Intercept Pharmaceuticals Inc.’s Ocaliva (obeticolic acid) and Genfit SA’s elafibranor could be the first products to be marketed for non-alcoholic steatohepatitis (NASH).
Ocaliva is the first drug to show it reduces fibrosis without worsening NASH in a Phase III study in non-cirrhotic NASH and liver fibrosis, the report says. The compound has the potential advantage of already being familiar to physicians as it is already approved for primary biliary cholangitis. However, it has been associated with increases in LDL-cholesterol and pruritus.
The Phase III RESOLVE-IT study of elafibranor in patients with NASH and fibrosis is due to readout at the end of 2019, possibly with a diagnostic which could help expand the NASH patient population, the report says. (Also see "Genfit Hopes To Make Case For First-Line Treatment In NASH" - Scrip, 4 Apr, 2019.)
Luspatercept In MDS
Acceleron Pharma's and Celgene’s luspatercept is involved in the later stages of erythropoiesis, while erythropoietin is involved in earlier stages, and is nearing the market for use in patients with myelodysplastic syndrome-associated anemia and beta-thalassemia-associated anemia.
In the MEDALIST Phase III study, 33.3% of MDS patients achieved transfusion independence for 12 weeks or more during the first 48 weeks of luspatercept treatment, compared with 11.8% of placebo-treated patients. Transfusion requirements were also reduced by the candidate therapy. Approval submissions have already been made in the US this year.
Horizon Pharma’s Teprotumumab
Active thyroid eye disease, also known as Graves’ ophthalmopathy, could gain its first pharmaceutical therapeutic with Horizon Pharma USA Inc.’s teprotumumab, which has reduced proptosis (eye bulging) in a high proportion of patients studied in the Phase III OPTIC trial, notes Biomedtracker.
An approval filing is planned for 2019 and because the product has US orphan drug, breakthrough therapy and fast-track designations, it could gain an approval early in 2020, with peak sales estimated at more than $750m. (Also see "Horizon Pharma Sees Mid-2019 BLA For Tepro After Solid Phase III TED Data" - Scrip, 1 Mar, 2019.)
Triplets In CF, Asthma
Vertex Pharmaceuticals Inc.'s triplet combination regimen involving a new next-generation corrector for cystic fibrosis, either VX-445 or VX-659 is nearing the market. Approval of the combination, which will include tezacaftor and ivacaftor, has been effective in patients with a wider range of genotypes, including those with difficult-to-treat CF.
The approval should be straightforward, but market access barriers may hold back the product’s commercial potential, Biomedtracker reckons. That said, the triple combination is expected to do well in large markets, like the US. (Also see "Cystic Fibrosis Triplet Could Unblock EU Reimbursement Impasse" - Scrip, 1 May, 2019.)
Another triple therapy in the works is the use of GlaxoSmithKline’s triple combination, Trelegy Ellipta, in the additional indication, severe asthma. Trelegy Ellipta contains an inhaled corticosteroid, long-acting beta2-agonist and long-acting muscarinic antagonist (fluticasone, umeclidinium and vilanterol), and are expected to be used in patients with exacerbations despite use of a dual combination of a steroid and a beta-agonist.
Further details of the Biomedtracker report, Key Potential Drug Launches in 2020, are available here.
Editor's Note: This article was updated on 13 June to reflect the fact that it relates to the US market and not to the first market overall.