March 2, 2017
By Alex Keown, BioSpace.com Breaking News Staff
CHICAGO – The orphan drug market faces scrutiny for high prices being charged to very small pools of patients. Despite those criticisms, multiple companies are developing drugs for orphan populations that, as John Carroll of Endpoints said, are offering patients “real hope for the first time.”
Citing a recent evaluation of the orphan drug market by Evaluate, Endpoints picked 10 orphan drug candidates that could, if approved, become blockbusters, or near blockbusters, by 2022.
1. Niraparib, Tesaro Inc.
Developed by Tesaro Inc. , Niraparib is a PARP inhibitor to treat ovarian cancer. Company data showed the drug could benefit 70 percent of all ovarian cancer patients who received previous chemotherapy treatments. Tesaro said niraparib increased progression free survival in different cohorts of a 553-patient trial—whether they had the BRCA gene or homologous recombination deficient (HRD) tumors. Trial data showed patients with the BRCA gene had 21 months of progression free survival—15 months longer than the control group. Additionally, patients in the non-germline BRCA mutant cohort showed progression free survival of 9.3 months, compared to 3.9 months for control. Evaluate estimates the drug, if approved, could generate $1.8 billion in sales by 2022.
2. Axicabtagene ciloleucel, Kite Pharma
Kite Pharma ’s CAR-T treatment in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL) will be presented to the U.S. Food and Drug Administration for approval soon. In recent trial data, the therapy reduced tumor sizes by half in more than 80 percent of dosed patients. According to study data, six months following the one infusion, 41 percent of patients achieved a positive tumor response, while 36 percent were in full remission. Evaluate estimates Kite’s CAR-T drug could generate $1.7 billion by 2022.
3. Emicizumab, Roche
Roche ’s hemophilia treatment earned the Breakthrough Therapy Designation from the U.S. Food and Drug Administration. The drug is a bispecific antibody engineered to bind both factors IXa and X, replacing the function of the missing factor VIII to improve clotting function and prevent spontaneous bleeding, according to Roche. Additionally, the therapy is designed to promote blood coagulation in hemophilia A patients, regardless of whether they have developed inhibitors to factor VIII. Evaluate estimates the drug could generate $1.5 billion in annual sales by 2022.
4. Tremelimumab, AstraZeneca
Tremelimumab is one of AstraZeneca ’s checkpoint inhibitors that is designed to work alongside durvalumab to treat head and neck squamous cell carcinoma. Tremelimumab inhibits the activity of cytotoxic T-lymphocyte-associated antigen-4 (prCTLA-4) to boost the immune response against cancer cells. Evaluate estimates that the drug could yield $1.3 billion in annual sales by 2022.
5. Lanadelumab, Shire
Shire ’s human monoclonal antibody is designed to prevent angioedema in patients with hereditary angioedema. Shire acquired the drug in 2015 with the acquisition of Dyax . During trials, Lanadelumab was able to show an 88 percent reduction in attacks. Evaluate estimates the drug could generate $1.1 billion annually by 2022, however Carroll said Shire executives believe the drug can generate $2 billion in annual sales.
6. SD-809, Teva
Earlier this week, the FDA granted priority review for Teva ’s SD-809, a treatment for tardive dyskinesia. The FDA is expected to rule on it in August. There are currently no approved treatments in the U.S. for tardive dyskinesia, a severe a movement disorder. Endpoints noted Teva’s SD-809 has already been rejected by the FDA once for Huntington’s cholera. Evaluate predicts if approved, SD-809 could yield $1 billion annually by 2022.
7. LentiGlobin, bluebird bio
bluebird bio ’s gene therapy is a treatment for sickle cell anemia and beta-thalassemia major. The company said early trial data showed treatment with the gene therapy proved positive for some patients with transfusion-dependent beta-thalassemia and one patient with severe sickle cell disease, were benefiting from the drug. The company is pursuing a Phase III trial, Endpoints said. If approved, Evaluate predicts it could yield $969 million in annual sales by 2022.
8. Acalabrutinib, AstraZeneca
AstraZeneca ’s second drug on the list, acalabrutinib is a second-generation, selective and potent inhibitor of Bruton’s tyrosine kinase (Btk). AstraZeneca is likely banking on the possibility of acalabrutinib becoming a blockbuster treatment for CLL, looking to compete with Venetoclax, a CLL treatment being jointly developed by AbbVie and Genentech . Evaluate predicts this treatment could generate $968 million in annual revenue by 2022.
9. CTL019, Novartis
Novartis ’s CAR-T treatment for pediatric leukemia 82 percent of patients’ blood cancer into remission. The global Phase II study found that 82 percent of pediatric patients, 41 out of 50, achieved complete remission or complete remission with incomplete blood count recovery at three months following infusion of CLT019. Novartis could see $917 million in annual sales from CTL019 by 2022, according to Evaluate.
10. Veliparib, AbbVie
AbbVie ’s PARP inhibitor received Orphan Drug Designation in November. Veliparib is being investigated in combination with chemotherapies, such as carboplatin and paclitaxel, or radiation for the treatment of advanced squamous non-small cell lung cancer (NSCLC). If approved, AbbVie could see $854 million in annual sales by 2022, according to Evaluate’s report.
