Insights+: The US FDA New Drug Approvals in August 2021

The US FDA has approved 3 BLAs and 2 NDAs in 2021, leading to treatments for patients and advances in the health care industry

The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 48 novel products in 2021

Additionally, last year in 2020, the US FDA has approved 121 novel products. PharmaShots has compiled a list of a total of 5 new drugs approved by the US FDA in August 2021

Sanofi’s Nexviazyme (avalglucosidase alfa-ngpt) Receives the US FDA’s Approval for the Treatment of Late-Onset Pompe Disease

Published: Aug 09, 2021 | Tags: Sanofi, Nexviazyme, avalglucosidase alfa-ngpt, US, FDA, Approval, Pompe Disease

• The approval is based on the P-III COMET trial that evaluates Nexviazyme (q2w, IV infusion) vs alglucosidase alfa in patients aged ≥1yrs. with LOPD for 49wks.
• Results: patients achieved noninferiority with 2.4 points greater improvement in FVC percent-predicted @49wks., were able to walk 32.2 m farther @49wks. on the 6MWT. The therapy showed improvements in respiratory function and walking distance with a safety profile
• Nexviazyme is an ERT targeting M6P receptor & previously received the FDA’s BTD & FTD for the treatment of PD. Additionally, the approval follows the FDA’s priority review granted in Nov’2018

Merck’s Welireg (belzutifan) Receives the US FDA’s Approval for the Treatment of Von Hippel-Lindau Disease Associated Tumors

Published: Aug 16, 2021 | Tags: Merck, Welireg, belzutifan, US, FDA, Approval, Von Hippel-Lindau Disease, Tumors

• The approval is based on open-label study 004 trial evaluates welireg (120 mg, qd) in patients with VHL-associated tumors including RCC, CNS hemangioblastomas, or pNET
• The results showed ORR (49%), m-DoR had not reached, 56% were still responding after 12mos., m-TTR (8mos.) in 61 patients with VHL-associated RCC, Additionally, 24 & 12 patients with CNS hemangioblastomas & pNET demonstrates ORR (63%/ 83%), m-DoR had not reached, CR rate (4% & 17%), PR rate (58% & 76%), patients were still responding (73% & 50%) after 12 mos., m-TTR was 3 & 8mos.
• Welireg is the first HIF-2α inhibitor therapy approved in the US and is expected to be available in early Sept’21

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Accelerated Approval for the Treatment of dMMR Recurrent or Advanced Solid Tumors

Published: Aug 18, 2021 | Tags: GSK, Jemperli, dostarlimab-gxly, US, FDA, Accelerated Approval, dMMR Recurrent, Solid Tumors

• The approval is based on the results from cohort A1 & F of the ongoing P-I GARNET trial evaluates dostarlimab (500mg, IV, q3w for 4 doses) as monothx. in 209 patients with dMMR solid tumors, including EC & non-endometrial cancer. The therapy was discovered by AnaptysBio & licensed to Tesaro
• Results: In all dMMR solid tumor, ORR (41.6%); CR rate (9.1%); PR rate (32.5%), m-DoR (34.7 mos.), 95.4% of patients maintain response for >6mos. Additionally, patients with non-endometrial cancer demonstrated an ORR (38.7%)
• The approval follows an FDA priority review of BLA. The therapy marks 2nd indication & is currently being evaluated in other studies as monothx. or in combination for other cancers

Cara and Vifor’s Korsuva (difelikefalin) Receive the US FDA’s Approval for the Treatment of Moderate-to-Severe Pruritus Due to CKD in Patients with Hemodialysis

Published: Aug 25, 2021 | Tags: Cara, Vifor, Korsuva, difelikefalin, US, FDA, Approval, Pruritus, CKD, Hemodialysis

• The approval is based on the two P-III trials i.e., KALM-1, KALM-2 along with additional 32 clinical studies that evaluate Korsuva vs PBO in patients with a mod. to sev. pruritus associated with CKD who undergoes hemodialysis
• Results: patients achieved four-point improvement from baseline on a measure of severe itch (40% & 37%) vs (21% & 26%) & was generally well tolerated
• Korsuva (inj.) is a KOR agonist targeting PNS & is expected to launch in the US in Q1’22 with reimbursement in H1’22. The NDA has received the US FDA’s priority review and if approved, the therapy will provide an improvement in the safety or effectiveness to treat serious conditions

Ascendis’ Skytrofa (lonapegsomatropin-tcgd) Receives the US FDA’s Approval for Pediatric Growth Hormone Deficiency

Published: Aug 27, 2021 | Tags: Ascendis, Skytrofa, lonapegsomatropin-tcgd, US, FDA, Approval, Growth Hormone Deficiency

• The approval is based on the P-III heiGHt trial evaluating Skytrofa (qw) vs somatropin in 161 treatment-naïve children ≥aged 1yrs. with GHD who have growth failure due to inadequate secretion of endogenous GH
• The study met its 1EPs i.e., non-inferiority in AHV & showed a higher AHV @52wks., no serious AEs or discontinuations related to Skytrofa were observed
• Skytrofa is the 1st product developed by using Ascendis’ TransCon technology platform & is being evaluated in P-III trials for pediatric GHD in Japan and Greater China. The therapy will be available in the US imminently by a full suite of patient support programs

Related Post: Insights+: The US FDA New Drug Approvals in July 2021