NEWLY FDA APPROVED DRUGS 2021- PART IV

As 2021 is coming to an end, it is important to look back at all of our accomplishments. Like many of us, the Food and Drug Administration (FDA) has been working tirelessly in efforts to improve the well being of many. In doing so, they have approved a total of 43 new drugs since the beginning of the year. Here are some of the most recent approvals:

1. KORSUVA– August 23

Cara Therapeutics and Vifor Pharma announced that the FDA has approved KORSUVA™ (difelikefalin) for injection for the treatment of moderate-to-severe pruritus associated with chronic kidney disease in adults undergoing hemodialysis. It is a first-in-class kappa opioid receptor (KOR) agonist that targets the body’s peripheral nervous system. KORSUVA received Priority Review by the FDA, which is granted to therapies that, if approved, would offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

2. SKYTROFA– August 25

SKYTROFA is a prescription medication for the replacement of growth hormone in children 1 year old or older who weigh at least 26 pounds (11.5 kilograms) with growth hormone deficiency (GHD). It is the first FDA approved treatment utilizing TransCon™ technology and is a once weekly, long-acting prodrug of somatropin that releases the same somatropin used in daily therapies. The approval includes the new SKYTROFA® Auto-Injector and cartridges which, after first removed from a refrigerator, allow families to store the medicine at room temperature for up to six months.

3. EXKIVITY– September 15

The FDA has approved EXKIVITY for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. EXKIVITY, which was granted priority review and received Breakthrough Therapy Designation, Fast Track Designation and Orphan Drug Designation from the FDA, is the first and only approved oral therapy specifically designed to target EGFR Exon20 insertion mutations.

4. TIVDAK– September 20

TIVDAK is a first-in-class antibody-drug conjugate for the treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. It is approved under the FDA’s Accelerated Approval Program based on tumor response and the durability of the response.

5. QULIPTA– September 28

QULIPTA is the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist specifically developed for the preventive treatment of episodic migraine. CGRP and its receptors are expressed in regions of the nervous system associated with migraine pathophysiology, and studies have shown that CGRP levels are elevated during migraine attacks. QULIPTA blocks CGRP through a once-daily dose and is available in three strengths – 10 mg, 30 mg and 60 mg.

6. LIVMARLI– September 29

LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome who are 1 year or older. ALGS is a rare genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease. Cholestatic pruritus is a sensation of itch associated with liver disease. The approval of LIVMARLI was based on findings from the pivotal ICONIC study and supported by 5 years of data from supportive studies in a total of 86 patients.

7. TAVENOS– October 7

TAVNEOS, an orally administered selective complement 5a receptor inhibitor, as an adjunctive treatment of adult patients with severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis, specifically granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA), in combination with standard therapy. ANCA-associated vasculitis is a systemic autoimmune disease in which over-activation of the complement system further activates neutrophils, leading to inflammation and eventual destruction of small blood vessels.

8. SCEMBLIX– October 29

SCEMBLIX has been approved for the treatment of chronic myeloid leukemia (CML) in two distinct indications. The FDA granted Scemblix accelerated approval for adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs), based on major molecular response (MMR) rate at 24 weeks; and full approval for adult patients with Ph+ CML-CP with the T315I mutation.

9. BESREMi– November 12

Back in 2009, BESREMi was approved for the treatment of a rare cancer disease that overproduces red blood cells, Polycythaemia Vera (PV), and other myeloproliferative neoplasms (MPNs) for European, Commonwealth of Independent States (CIS), and Middle Eastern markets. Finally, it has been approved by the FDA for US use. Besides achieving high rates of complete hematologic responses including freedom of phlebotomy in 8 out of 10 patients, BESREMi offers the possibility of disease modification and eventually operational cure in a subset of patients.

A huge thank you to everyone at the FDA who have pushed through these approvals despite the current state of our country. These approvals will create a better means of life for many. For more information on past approvals, visit the FDA website.