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

The US FDA has approved 5 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 55 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 7 new drugs approved by the US FDA in September 2021

Impel NeuroPharma’s Trudhesa (dihydroergotamine mesylate) Nasal Spray Receives the US FDA’s Approval for the Treatment of Migraine

Published: Sept 07, 2021 | Tags: Impel NeuroPharma, Trudhesa, dihydroergotamine mesylate, Nasal Spray, US, FDA, Approval, Migraine

• The approval is based on the P-III STOP 301 trial evaluates Trudhesa (0.725 mg/spray) in 5650+ patients with migraine with or without aura for 24 or 52 wks.
• The exploratory efficacy results showed that nasal spray provides rapid, sustained, and consistent symptom relief. The therapy is well-tolerated & no serious TEAEs were observed
• Trudhesa is the first and only therapeutic to use Impel’s pod technology to deliver DHE to the vascular-rich upper nasal space. The product is expected to launch in early Oct’21 & will be available through Trudhesa Direct and the copay program

Takeda’s Exkivity (mobocertinib) Receives the US FDA’s Approval as the First Oral Therapy for EGFR Exon20 Insertion+ NSCLC

Published: Sept 16, 2021 | Tags: Takeda, Exkivity, mobocertinib, US, FDA, Approval, EGFR Exon20 Insertion+ NSCLC

• The approval is based on the P-I/II trial evaluating Exkivity (160 mg) in 114 patients with EGFR Exon20 insertion+ NSCLC who received prior Pt-based therapy
• The results demonstrated ORR (28%) as assessed by IRC & 35% as per investigator, m-DoR (17.5mos.), m-OS (24mos.), and m-PFS (7.3mos.) as per IRC
• Exkivity is a TKI inhibitor designed to selectively target EGFR Exon20 insertion mutation & has received PR, BTD, FTD & ODD from the FDA. Additionally, the US FDA has approved Thermo Fisher’s Oncomine Dx Target test as an NGS CDx for Exkivity to identify NSCLC patients with EGFR Exon20 insertions

Samsung Bioepis and Biogen’s Byooviz (biosimilar, ranibizumab) Receives the US FDA’s Approval for the Treatment of Retinal Vascular Disorders

Published: Sept 21, 2021 | Tags: Samsung Bioepis, Biogen, Byooviz, biosimilar, ranibizumab, US, FDA, Approval, Retinal Vascular Disorders

• The approval is based on a totality of evidence including analytical, non-clinical/clinical data from the P-III study evaluates the efficacy, safety, PK & immunogenicity of SB11 (0.5mg) vs Lucentis in a ratio (1:1) in 705 patients with wet AMD
• The results showed LS mean change in BCVA from baseline @52wks. (9.79 vs 10.41 letters) & LS mean change in CST (−139.55 vs −124.46 μm) while PK, safety including the incidence of TEAEs & immunogenicity profile were comparable at all timepoints @52wks.
• Byooviz marks the 1st ophthalmology biosimilar approved in the US. In Nov’19, Samsung Bioepis & Biogen had entered into a commercialization agreement for 2 ophthalmology biosimilar i.e., SB11 & SB15

Incyte’s Opzelura (ruxolitinib) Receives the US FDA’s Approval for the Treatment of Atopic Dermatitis

Published: Sept 22, 2021 | Tags: Incyte, Opzelura, ruxolitinib, US, FDA, Approval, Atopic Dermatitis

• The approval is based on the TRuE-AD clinical trial program which consists of two P-III studies i.e., TRuE-AD1 & 2 evaluating the safety and efficacy of Opzelura cream (1.5%, bid) vs vehicle (non-medicated cream) in 1200+ adolescents & adults aged ≥12yrs. with mild to moderate AD
• The results showed that the patients achieved IGA-TS @8wks. (53.8% & 51.3% vs 15.1% & 7.6%) & clearer skin, reduction in itch from baseline @8wks. as measured by a ≥4-point reduction in the itch NRS4 (52.2% & 50.7% vs 15.4% & 16.3%) respectively
• Opzelura is the first topical formulation of a JAK inhibitor approved in the US. The therapy is currently being evaluated is in the P-III TRuE-V clinical program for the treatment of vitiligo

Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv) Receive the US FDA’s Accelerated Approval for the Treatment of Recurrent or Metastatic Cervical Cancer

Published: Sept 22, 2021 | Tags: Seagen, Genmab, Tivdak, tisotumab vedotin-tftv, US, FDA, Accelerated Approval, Recurrent, Metastatic Cervical Cancer

• The approval is based on the P-II innovaTV 204 clinical trials evaluating tisotumab vedotin in 101 patients with r/mCC who had received no more than two prior systemic regimens in the recurrent or metastatic setting, including one prior Pt-based CT regimen
• The results showed an ORR (24%) as assessed by IRC using RECIST v1.1 criteria, m-DoR (8.3mos.)
• Tivdak is the first approved ADC that directed to TF and Seagen’s ADC technology that utilizes a protease-cleavable linker & covalently attaches the microtubule-disrupting agent MMAE to Ab. The P-III innovaTV 301clinical trial of Tivdak is underway that is intended to support global registrations

AbbVie’s Qulipta (atogepant) Receives the US FDA’s Approval for the Treatment of Migraine

Published: Sept 29, 2021 | Tags: AbbVie, Qulipta, atogepant, US, FDA, Approval, Migraine

• The approval was based on a clinical program including P-III ADVANCE study, P-IIb/III study & P-III LTS study evaluated the safety, efficacy & tolerability of Qulipta (PO, 10/30/60mg) vs PBO in 2000 patients with EM who experienced 4-14 migraine days/mos.
• The P-III ADVANCE & P-IIb/III study met its 1EPs i.e., reduction in mean monthly migraine days @12wks. for all 3 doses. The 2EPs of the P-III trial showed that the proportion of patients achieved a 50-100% reduction in monthly migraine days (56%/59%/61% vs 29.0%) @12wks. & all doses were well tolerated in both studies
• In the P-III study, patients treated with 60mg dose experienced a 4.2-day reduction from a baseline of 7.8 @12wks.

Mirum’s Livmarli (maralixibat) Receives US FDA’s Approval as the First Approved Therapy for Cholestatic Pruritus in Patients with Alagille Syndrome

Published: Sept 29, 2021 | Tags: Mirum, Livmarli, maralixibat, US, FDA, Approval, Cholestatic Pruritus, Alagille Syndrome

• The approval is based on the ICONIC study as well as 5yrs. of data from supportive studies that evaluate Livmarli in 86 patients with ALGS
• The results from the ICONIC study demonstrated a significant reduction in pruritus that was maintained through 4yrs.
• Livmarli is the first FDA-approved therapy for patients aged ≥1yrs. & & the company has received a rare pediatric disease priority review voucher. The therapy will now be accessible to patients with a prescription through MAP that provides insurance coverage, and access support along with financial support options for patients with Livmarl

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