Top 10 Selling Orphan Drugs
AbbVie/J&J’s IMBRUVICA (ibrutinib) top the table, the drug which is a bruton’s tyrosine kinase (BTk) inhibitor used to treat,
- Adults with chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL)
- Adults with chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL) with 17p deletion
- Adults with Waldenström’s macroglobulinemia (WM)
- Adults and children 1 year of age and older with chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy
J&J’s DARZALEX is used to treat adults with multiple myeloma,
- In combination with the medicines lenalidomide and dexamethasone in people with newly diagnosed multiple myeloma who cannot receive a type of stem cell transplant that uses their own stem cells (autologous stem cell transplant) and in people whose multiple myeloma has come back or did not respond to treatment who have received at least one prior medicine to treat multiple myeloma
- In combination with the medicines bortezomib, melphalan, and prednisone in people with newly diagnosed multiple myeloma who cannot receive a type of stem cell transplant that uses their own stem cells (autologous stem cell transplant)
- In combination with the medicines bortezomib, thalidomide, and dexamethasone in newly diagnosed people who are eligible to receive a type of stem cell transplant that uses their own stem cells (autologous stem cell transplant)
- In combination with the medicines bortezomib and dexamethasone in people who have received at least one prior medicine to treat multiple myeloma
- In combination with the medicines carfilzomib and dexamethasone in people whose multiple myeloma has come back or did not respond to treatment who have received one to three prior medicines to treat multiple myeloma
- In combination with the medicines pomalidomide and dexamethasone in people who have received at least two prior medicines to treat multiple myeloma, including lenalidomide and a proteasome inhibitor
- Alone in people who have received at least three prior medicines, including a proteasome inhibitor and an immunomodulatory agent, or did not respond to a proteasome inhibitor and an immunomodulatory agent
Vertex Pharma’ TRIKAFTA is a combination of ivacaftor, a CFTR potentiator, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data.
Roche’s HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors.
AstraZeneca’ LYNPARZA used to treat adults who have:
- Advanced ovarian cancer, fallopian tube cancer, or primary peritoneal cancer with a certain type of inherited (germline) or acquired (somatic) abnormal BRCA gene
- Advanced ovarian cancer, fallopian tube cancer, or primary peritoneal cancer with a certain type of abnormal BRCA gene or a positive laboratory tumor test for genomic instability called HRD
- In combination with another anti-cancer medicine, bevacizumab, as maintenance treatment after the cancer has responded to your first treatment with platinum-based chemotherapy
- Ovarian cancer, fallopian tube cancer, or primary peritoneal cancer with a certain type of inherited (germline) or acquired (somatic) abnormal BRCA gene. LYNPARZA is used as maintenance treatment when the cancer has come back
- When the cancer has responded to treatment with platinum-based chemotherapy
- For human epidermal growth factor receptor 2 (HER2)-negative early breast cancer with a certain type of inherited (germline) abnormal BRCA gene.
- After surgery (treatment after surgery is called adjuvant therapy), a certain type of abnormal inherited BRCA gene, human epidermal growth factor receptor 2 (HER2)-negative breast cancer that has spread to other parts of the body (metastatic).
- Metastatic pancreatic cancer with a certain type of abnormal inherited BRCA gene. Used as a maintenance treatment after your cancer has not progressed on at least 16 weeks of treatment with platinum-based chemotherapy.
- Prostate cancer with certain inherited or acquired abnormal genes called homologous recombination repair (HRR genes). When the cancer has spread to other parts of the body (metastatic), and no longer responds to a medical or surgical treatment that lowers testosterone, and has progressed after treatment with enzalutamide or abiraterone.
- Prostate cancer with a certain type of abnormal inherited or acquired BRCA gene that has spread to other parts of the body (metastatic) and no longer responds to a medical or surgical treatment that lowers testosterone. Used in combination with another anti-cancer medicine, abiraterone, together with the steroid medicine, prednisone or prednisolone
Incyte’s JAKAFI is a prescription medicine used to treat adults with polycythemia vera who have already taken a medicine called hydroxyurea and it did not work well enough or they could not tolerate it.
- To treat adults with certain types of myelofibrosis.
- To treat adults and children 12 years of age and older with acute graft-versus-host disease (GVHD) who have taken corticosteroids and they did not work well enough.
- To treat adults and children 12 years of age and older with chronic GVHD who have taken one or two types of treatments and they did not work well enough.
AstraZeneca’s ULTOMIRIS is currently approved for the treatment of certain adults with NMOSD in the European Union (EU), Japan and other countries.
- Ultomiris is approved by the FDA for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) Ab+, and certain adults and children with paroxysmal nocturnal haemoglobinuria (PNH) or atypical haemolytic uraemic syndrome (aHUS).
AbbVie’s VENCLEXTA is a prescription medicine used:
- To treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- In combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly diagnosed acute myeloid leukemia (AML) who: are 75 years of age or older, or have other medical conditions that prevent the use of standard chemotherapy.
AstraZeneca’s CALQUENCE is a prescription medicine used to treat adults with mantle cell lymphoma (MCL) who have received at least one prior treatment for their cancer, or adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Pfizer’s VYNDAQEL are transthyretin stabilizers indicated for the treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.
