Oncology Drug Approvals in 2020

Though 2020 was a challenging year for all of us, there were some good news for many cancer patients. In oncology, the U.S. Food and Drug Administration (FDA) granted approval to several novel drugs and new indications for older therapeutic agents. According to the FDA’s count, it approved as many as 53 new molecular entities in 2020 beating the previous year which saw 48 new molecular entities approved. Several new oncology drug approvals are for patient groups with previously unmet therapeutic needs and rare disease groups. Let us look back at 10 key FDA drug approvals from 2020 in the oncology space, as determined by the Analysis Team at PubGene AS. 

No.Drug NameCompanyDateApproved Use
1Avyakit
(avapritnib)
Blueprint
Medicines
09/01Unresectable or metastatic
gastrointestinal stromal tumor
2Tazverik
(tazemetostat)
Epizyme23/01Epithelioid sarcoma
3Sarclisa
(isatuximab)
Sanofi Aventis02/03Multiple myeloma
4Tukysa
(tucatinib)
Seattle
Genetics
17/04Advanced un-resectable or metastatic HER-2 positive breast cancer
5Pemazyre
(pemigatinib)
Incyte17/04Cholangiocarcinoma
6Trodelvy
(sacituzumab
govitecan-hziy)
Immunomedics22/04Metastatic triple-negative breast
cancer who received at least two prior therapies for metastatic disease
7Tabrecta
(capmatinib)
Novartis06/05Non-small cell lung cancer
8Retevmo
(selpercatinib)
Loxo Oncology08/05Lung and thyroid cancer
9Qinlock
(repretinib)
Deciphera15/05Advanced gastro-intestinal tumors
10Orgovyk
(relugolix)
Myovant
Sciences, Inc.
18/12Advanced prostate cancer

1. Ayvakit (Avapritinib)

Ayvakit is a type of targeted therapy called a tyrosine kinase inhibitor. It works by blocking certain proteins (including PDGFRA) that help the cancer cells divide and grow. About 5% to 10% of gastrointestinal stromal tumor (GIST)s have a PDGFRA mutation. These tumors usually do not respond well to therapies already on the market. Ayvakit is the first therapy approved for patients with GIST harboring a PDGFRA exon 18 mutation. Read more here.

2. Tazverik (Tazemetostat)

Tazverik is a small molecule that acts as an inhibitor of the histone methyltransferase Enhancer of Zeste Homologue 2 (EZH2). Histone methyltransferases are enzymes that can suppress (turn off) the activity of specific genes. EZH2 blocks the expression of tumor suppressor genes in healthy cells. Tumor suppressors can keep the cells from dividing too quickly, repair DNA mistakes, or tell cells when to die. The USFDA has approved Tazverik (tazemetostat) to treat adults and children 16 and older with epithelioid sarcoma, a rare cancer which accounts for less than 1% of all soft tissue sarcoms. Tazverik is only the second targeted therapy approved for a soft tissue sarcoma and the first treatment option specifically for epithelioid sarcoma Read more here.

3. Sarclisa (Isatuximab-irfc)

Though 2020 was a challenging year for all of us, there were some good news for many cancer patients. In oncology, the U.S. Food and Drug Administration (FDA) granted approval to several novel drugs and new indications for older therapeutic agents. According to the FDA’s count, it approved as many as 53 new molecular entities in 2020 beating the previous year which saw 48 new molecular entities approved. Several new oncology drug approvals are for patient groups with previously unmet therapeutic needs and rare disease groups. Let us look back at 2020’s 10 key FDA drug approvals in the oncology space, as determined by the Analysis Team at PubGene AS. 

4. Tukysa (Tucatinib)

Tukysa is a tyrosine kinase inhibitor that blocks the HER2 receptor. Around 20% of breast tumors express this receptor at high levels and can be treated with HER2 inhibitors such as Herceptin (trastuzumab), Perjeta (pertuzumab) or Kadcyla (ado-trastuzumab emtansine). FDA approved Tukysa as part of a combination regimen for people with previously treated inoperable or metastatic HER2-positive breast cancer, including cancer that has spread to the brain. This approval represents an additional targeted treatment option for the many patients with HER2-positive breast cancer. Read more here.

5. Pemazyre (Pemigatinib)

Pemazyre is a tablet that works by blocking fibroblast growth factor receptor 2 (FGFR2) in tumor cells to prevent them from growing and spreading. USFDA granted accelerated approval to Pemazyre, the first targeted treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma, a cancer of bile ducts. FGFR2 fusions have been found in the tumors of approximately 9% to 14% of patients with cholangiocarcinoma. Pemazyre is a new option for these patients following first line with chemotherapy. Read more here.

6. Trodelvy

Trodelvy is a Trop-2-directed antibody and topoisomerase inhibitor drug conjugate, meaning that the antibody part of the drug targets the Trop-2 receptor that helps the cancer grow, divide and spread, and is linked to a topoisomerase inhibitor, which is toxic to cancer cells. Approximately two of every 10 breast cancer diagnoses worldwide are triple-negative. The approval of Trodelvy represents a new targeted therapy for patients with triple-negative breast cancer that has spread to other parts of the body and who have tried at least two prior therapies. Read more here.

7. Tabrecta (Capmatinib)

Tabrecta is a kinase inhibitor, meaning it functions by blocking a key enzyme that results in helping to stop the tumor cells from growing. Tabrecta is the first FDA-approved therapy to treat non-small cell lung cancer (NSCLC) with specific mutations (those that lead to mesenchymal-epithelial transition or MET exon 14 skipping). NSCLC is the most common type of lung cancer with up to 90% of all lung carcinomas falling into the non-small cell category. Cancer metastasis consists of a sequential series of events and MET exon 14 skipping is recognized as a critical event for metastasis of carcinomas. Mutations leading to MET exon 14 skipping are found in 3-4% of patients with lung cancer. Read more here.

8. Retevmo (Selpercatinib)

Retevmo is a kinase inhibitor, meaning it blocks a type of enzyme (kinase) and helps prevent the cancer cells from growing. Before beginning treatment, the identification of a RET gene alteration must be determined using laboratory testing. USFDA approved Retevmo capsules to treat three types of tumors – non-small cell lung cancer, medullary thyroid cancer and other types of thyroid cancers – in patients whose tumors have an alteration (mutation or fusion) in a specific gene (RET or “rearranged during transfection”). Retevmo is the first therapy approved specifically for cancer patients with the RET gene alterations. Read more here.

9. Quinlock (Ripretinib)

Qinlock is a kinase inhibitor, meaning it works by blocking a type of enzyme called a kinase, which helps keep the cancer cells from growing. Despite the progress that has been made over the past 20 years in developing treatments for GIST, including four FDA-approved targeted therapies – imatinib in 2002, sunitinib in 2006, regorafenib in 2013 and avapritinib earlier this year – some patients don’t respond to treatment and their tumors continues to progress. Qinlock’s approval provides a new treatment option for patients who have exhausted all FDA-approved therapies for GIST. Read more here.

10. Orgovyx (Relugolix)

FDA approved the first oral gonadotropin-releasing hormone (GnRH) receptor antagonist, Orgovyx for adult patients with advanced prostate cancer. Existing FDA-approved treatments for advanced prostate cancer are injected or placed as small implants under the skin. Orgovyx is an orally administered treatment that works by blocking the pituitary gland from making hormones called luteinizing hormone and follicle-stimulating hormone, thereby reducing the amount of testosterone the testicles are able to make. This can help patients with cancer stay home and avoid exposure during the coronavirus pandemic. Read more here.

For complete prescribing information for any of these agents, visit www.accessdata.fda.gov/scripts/cder/daf.