Breakthrough therapy

Sebelipase alfa is a recombinant form of the enzyme lysosomal acid lipase (LAL) that was approved in 2015 under the tradename Kanuma in the US and EU for the treatment of people with lysosomal acid lipase deficiency (LAL-D).

Ledipasvir (formerly GS-5885) is a drug for the treatment of hepatitis C that was developed by Gilead Sciences.

Idelalisib (trade name Zydelig zye-DEL-ig, codenamed GS-1101 or CAL-101) is a drug used for the treatment of certain hematological malignancies.

Sirolimus (INN/USAN), also known as rapamycin, is a macrolide compound produced by the bacterium Streptomyces hygroscopicus that is used in medicine to prevent organ transplant rejection and to treat lymphangioleiomyomatosis.

Ranibizumab (trade name Lucentis among others) is a monoclonal antibody fragment (Fab) created from the same parent mouse antibody as bevacizumab.

Sofosbuvir (brand name Sovaldi) is a nucleotide analog used in combination with other drugs for the treatment of hepatitis C virus (HCV) infection.

Ibrutinib (USAN, also known as PCI-32765 and marketed under the name Imbruvica) is an anticancer drug targeting B-cell malignancies.

Ofatumumab (trade name Arzerra, also known as HuMax-CD20) is a fully human monoclonal antibody (for the CD20 protein) which appears to inhibit early-stage B lymphocyte activation.

Ritonavir, sold under the trade name Norvir, is an antiretroviral medication used along with other medications to treat HIV/AIDS This combination treatment is known as highly active antiretroviral therapy (HAART).

Ombitasvir is an antiviral drug for the treatment of hepatitis C virus (HCV) infection.

Paritaprevir (previously known as ABT-450) is an acylsulfonamide inhibitor of the NS3-4A serine protease manufactured by Abbott Laboratories that shows promising results as a treatment of hepatitis C.

Pirfenidone (INN, BAN) is an anti-fibrotic drug for the treatment of idiopathic pulmonary fibrosis (IPF).

Pembrolizumab (formerly MK-3475 and lambrolizumab, trade name Keytruda) is a humanized antibody used in cancer immunotherapy.

Alectinib (marketed as Alecensa) is an oral drug that blocks the activity of anaplastic lymphoma kinase (ALK) and is used to treat non-small cell lung cancer (NSCLC).

Nintedanib, marketed under the brand names Ofev and Vargatef, is a medication used for the treatment of idiopathic pulmonary fibrosis (IPF) and along with other medications for some types of non-small-cell lung cancer.

Blinatumomab (trade name Blincyto, previously known as AMG103 and MT103) is a biopharmaceutical drug used as a second-line treatment for Philadelphia chromosome-negative relapsed or refractory acute lymphoblastic leukemia.

Daratumumab (Darzalex) is an anti-cancer drug.

Elotuzumab (brand name Empliciti, previously known as HuLuc63) is a humanized monoclonal antibody used in relapsed multiple myeloma.

Obinutuzumab (called afutuzumab until 2009, originally GA101) is a humanized anti-CD20 monoclonal antibody, originated by GlycArt Biotechnology AG and developed by Roche as a cancer treatment.

Osimertinib (previously known as mereletinib or AZD9291; trade name Tagrisso) is a third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) drug developed by AstraZeneca Pharmaceuticals – for mutated EGFR cancers.

Lumacaftor/ivacaftor (brand name Orkambi) is a combination drug available as a single pill that is used for the treatment of cystic fibrosis in people who have two copies of the F508del mutation in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein.

Aflibercept (INN, USAN) is a biopharmaceutical drug invented by Regeneron Pharmaceuticals, approved in the United States and Europe for the treatment of wet macular degeneration under the trade name Eylea, and for metastatic colorectal cancer as Zaltrap.

Ceritinib (trade name Zykadia) is a drug for the treatment of a specific type of lung cancer.

Nivolumab (nye vol' ue mab), marketed as Opdivo, is a human IgG4 anti-PD-1 monoclonal antibody used to treat cancer.

Palbociclib (codenamed PD-0332991, trade name Ibrance) is a drug for the treatment of ER-positive and HER2-negative breast cancer developed by Pfizer.

Eltrombopag (rINN, codenamed SB-497115-GR) is a medication that has been developed for certain conditions that lead to thrombocytopenia (abnormally low platelet counts).

A combination product of three antiviral drugs ombitasvir, paritaprevir and ritonavir is used with the drug dasabuvir in the treatment of hepatitis C Virus Genotype 1a or 1b (HCV GT1a or GT1b).

Ivacaftor (trade name Kalydeco, developed as VX-770) is a drug used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, who account for 4–5% cases of cystic fibrosis, and is included in a combination drug, lumacaftor/ivacaftor, which is used to treat people with cystic fibrosis who have the F508del mutation in CFTR.