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

The US FDA has approved 4 NDAs and 2 BLAs 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 61 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 October 2021

Kite’s Tecartus Receives the US FDA’s Approval as the First and Only Car T for Adults with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

Published: Oct 04, 2021 | Tags: Kite, Tecartus, US, FDA, Approval, Car T, B-cell Acute Lymphoblastic Leukemia

• The approval was based on a P-I/II ZUMA-3 study assessing Tecartus in adult patients aged ≥18yrs. with ALL whose disease is refractory to or has relapsed following first standard systemic therapy with remission of 12mos. or less, after 2L+ systemic therapy or at least 100 days after ASCT
• Results: 65% of the evaluable patients achieved CR or CRi @median actual follow-up of 12.3 mos.; mDOR (13.6mos.); Grade 3 or higher CRS (26%); neurologic events (35%); well tolerated
• Tecartus is the first and only CAR T-cell therapy approved for adults aged ≥18yrs. with ALL. The approval marks Kite’s fourth indication for its cell therapies and first in Leukemia

ChemoCentryx’s Tavneos (avacopan) Receives the US FDA’s Approval as an Adjunctive Treatment for Adult Patients with ANCA-Associated Vasculitis

Published: Oct 08, 2021 | Tags: ChemoCentryx, Tavneos, avacopan, US, FDA, Approval, ANCA-Associated Vasculitis

• The approval is based on the P-III ADVOCATE trial that evaluates Tavneos in 330 patients with ANCA-associated vasculitis across 20 countries. Patients were randomized to receive either rituximab or cyclophosphamide & Tavneos or prednisone
• The study met its 1EPs of disease remission @26wks. and sustained remission @52wks. as assessed by BVAS. The study also showed superiority to a prednisone-based SoC for sustained remission
• Avneos is a selective complement 5a receptor inhibitor & is also approved to treat MPA & granulomatosis with polyangiitis in Japan. The regulatory decision in the EU follows the EMA review which is expected at the end of 2021

Genentech’s Susvimo Receives the US FDA’s Approval for the Treatment of Wet Age-Related Macular Degeneration

Published: Oct 25, 2021 | Tags: Genentech, Susvimo, US, FDA, Approval, Wet Age-Related Macular Degeneration

• The approval is based on P-III Archway study evaluates Susvimo (100 mg/mL, IV, q3mos.) via ocular implant vs ranibizumab (0.5mg, IV) in 415 patients with wet AMD prior responded to 2 anti- VEGF
• The results showed that the therapy achieved & maintained vision gains equivalent to ranibizumab @ 36 & 40wks.; 1.6% of patients received supplemental ranibizumab treatment & 98% can go 6mos. before 1st refill. The therapy was well-tolerated with a favorable benefit-risk profile
• Susvimo is expected to be available in the US in the coming mos. & is also currently under EMA’s review for wet AMD. The company plans to provide patient assistance programs through Genentech access solutions

Bausch + Lomb and Clearside’s Xipere Receive the US FDA’s Approval for the Treatment of Macular Edema Associated with Uveitis

Published: Oct 29, 2021 | Tags: Bausch + Lomb, Clearside, Xipere, US, FDA, Approval, Macular Edema, Uveitis

• The approval is based on the P-III PEACHTREE trial to evaluate Xipere in 160 patients with macular edema associated with uveitis. The therapy is expected to be available in Q1’22
• The results showed that the greater proportion of patients treated with Xipere had achieved an improvement in BCVA (47% vs 16%) @24wks. Additionally, safety and efficacy data of Xipere had shown in multiple clinical studies
• Xipere is the 1st therapy to be available in the US & is designed to treat macular edema associated with uveitis via suprachoroidal administration using SCS Microinjector which has been developed by Clearside

Novartis’ Scemblix (asciminib) Receives the US FDA’s Approval for the Treatment of Chronic Myeloid Leukemia

Published: Oct 29, 2021 | Tags: Novartis, Scemblix, asciminib, US, FDA, Approval, Chronic Myeloid Leukemia

• The approval is based on P-III ASCEMBL & P-I study that evaluates Scemblix in patients with Ph+ CML-CP with T315I mutation who had experienced resistance or intolerance to two TKIs
• The P-III ASCEMBL trial showed an MMR rate (25% vs 13%) @24wks. & patients who discontinued treatment due to AEs (7% vs 25%). An additional P-I data in patients with CML with T315I mutation support the US FDA’s approval for 2nd indication in this patient population
• Scemblix is the 1st FDA-approved CML treatment that binds to ABL myristoyl pocket & has received the US FDA’s accelerated & full approval for Ph+ CML-CP. The company has initiated regulatory submission for Scemblix in multiple countries globally

Allergan’s Vuity Receives the US FDA’s Approval as First Eye Drop for the Treatment of Presbyopia

Published: Oct 29, 2021 | Tags: Allergan, Vuity, US, FDA, Approval, Presbyopia

• The approval is based on the P-III GEMINI 1 & 2 studies to evaluate the efficacy, safety, and tolerability of Vuity vs PBO in a ratio (1:1) in 750 patients aged 40 to 55yrs. with presbyopia
• The studies met its 1EPs i.e., improvement in near vision in low light (mesopic) conditions without impacting distance vision on 30 at 3hrs., improvement was observed as early as 15min. through 6hrs. The therapy was safe, well-tolerated, and effective for managing age-related blurry near vision & no serious AEs were observed in both studies
• Vuity (pilocarpine HCI ophthalmic solution) is the first FDA-approved eye drop to treat a progressive eye condition

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