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

The US FDA has approved 6 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 43 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 6 new drugs approved by the US FDA in July 2021

Bayer’s Kerendia (finerenone) Receives the US FDA’s Approval for the Treatment of Chronic Kidney Disease Associated with Type 2 Diabetes

Published: July 12, 2021 | Tags: Bayer, Kerendia, finerenone, US, FDA, Approval, Chronic Kidney Disease, Type 2 Diabetes

• The approval is based on a P-III FIDELIO-DKD study evaluating finerenone (10/20mg) + SoC vs PBO + SoC in ~ 5700 patients with CKD & T2D. Patients were followed for a median of 2.6yrs.
• Results: reduction in the incidence of the primary composite EPs of a sustained decline in eGFR of ≥40%, reduction in kidney failure, renal death, CV death, non-fatal MI, non-fatal stroke, or hospitalization for HF
• Finerenone has been submitted for marketing authorization in the EU & China along with multiple other countries globally & is currently under review. The therapy is expected to be available in the US at the end of July’21

Sanofi’s Fexinidazole Receives the US FDA’s Approval as the First Oral Treatment for Sleeping Sickness

Published: July 19, 2021 | Tags: Sanofi, Fexinidazole, US, FDA, Approval, Sleeping Sickness

• The US FDA has approved fexinidazole (PO, qd, 10days treatment) for both stages of Trypanosoma brucei gambiense form of sleeping sickness in patients aged >6yrs. & weighing at least 20 kg. The therapy has developed under the collaboration with DNDi, DRC, CAR & Sanofi
• Sanofi & partners are committed to access fexinidazole in all endemic countries for sleeping sickness because current treatment options for sleeping sickness are effective but troublesome for patients & health workers who live in remote areas
• Following the approval, Tropical disease PRV has been awarded to DNDi. Sanofi is committed to provide drug free-of-charge to WHO for distribution affected countries

Kadmon’s Rezurock (belumosudil) Receives the US FDA’s Approval for the Treatment of Chronic Graft Versus Host Disease

Published: July 20, 2021 | Tags: Kadmon, Rezurock, belumosudil, US, FDA, Approval, Chronic Graft Versus Host Disease

• The approval is based on KD025-213 study evaluating Rezurock (200 mg, qd) aged >12 yrs. in 65 patients with cGVHD who had received 2 to 5 prior lines of systemic therapy. The anticipated PFUFA date is Aug 30, 2021
• Results: ORR (75%) through Cycle 7, day 1 treatment with CR (6%) & PR (69%); median time to 1st response (1.8mos.), 62% of responders did not require new systemic therapy for at least 12mos. following response, m-DoR (1.9mos.), a median time from cGVHD diagnosis was 25.3 mos.
• Rezurock (belumosudil) is the 1st approved therapy targeting ROCK2 and expected to be available in the US in Aug’21. The FDA has granted BTD & PR for Rezurock & reviewed the NDA under RTOR pilot program

Albireo’s Bylvay (odevixibat) Receives the US FDA’s Approval for the Treatment of Progressive Familial Intrahepatic Cholestasis

Published: July 21, 2021 | Tags: Albireo, Bylvay, odevixibat, US, FDA, Approval, Progressive Familial Intrahepatic Cholestasis

• The approval is based on P-III PEDFIC 1 & 2 studies evaluating Bylvay (qd) vs PBO in patients with pruritus in PFIC. The PEDFIC 1 study met its 1EPs i.e the therapy showed a low incidence of diarrhea or frequent bowel movements (9.5% vs 5.0%)
• The PEDFIC 2 st1udy showed sustained reductions in serum bile acids & improvements in pruritus assessments, growth & other markers of liver function @ 48 wks. & well tolerated across both studies
• Bylvay is expected to be launch immediately in the coming days & is also being evaluated in a P-III BOLD trial for biliary atresia & P-III ASSERT trial for Alagille syndrome while results from the BOLD & ASSERT trial is expected in 2024 & 2022

Sol-Gel Technologies’ Twyneo (tretinoin/benzoyl peroxide) Cream Receives the US FDA’s Approval for the Treatment of Acne Vulgaris

Published: July 27, 2021 | Tags: Sol-Gel Technologies, Twyneo, tretinoin, benzoyl peroxide, Cream, US, FDA, Approval, Acne Vulgaris

• The approval is based on two P-III studies that evaluate Twyneo vs vehicle in adults & pediatric patients aged ≥9yrs. with acne vulgaris which demonstrated an efficacy & a favorable tolerability profile
• Twyneo utilizes Sol-Gel’s microencapsulation technology to entraps tretinoin & benzoyl peroxide within silica-based microcapsules. Additionally, the company collaborated with Galderma to commercialize Twyneo in the US
• Sol-Gel to receive a regulatory milestone in conjunction with FDA’s approval & retains the option to regain US commercialization rights for five yrs. after the 1st commercialization in the US. Twyneo is patent protected until 2038

Janssen’s Uptravi (selexipag) Receives the US FDA’s Approval for IV Use in Adults with Pulmonary Arterial Hypertension

Published: July 30, 2021 | Tags: Janssen, Uptravi, selexipag, US, FDA, Approval, Pulmonary Arterial Hypertension

• The approval is based on P-III UPTRAVI IV study evaluating the safety, tolerability, and PK of temporarily switching Uptravi (PO) to Uptravi (IV) in 20 patients with PAH
• The results showed that switching b/w Uptravi (PO) to Uptravi IV was well tolerated with no unexpected safety findings. Both formulations maintain the treatment effect for short-term temporary interruptions, AEs from Uptravi IV were similar to those associated with Uptravi tablets
• Uptravi is a selective, prostacyclin IP receptor agonist, approved for IV use for PAH, WHO Group I in the adult with WHO (FC) II–III, who are temporarily unable to take oral therapy

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