The US FDA has approved 4 NDAs and 1 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 29 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 May 2021

Apellis Empaveli (pegcetacoplan) Receives the US FDA's Approval for Paroxysmal Nocturnal Hemoglobinuria

Published: May 17, 2021 | Tags: Apellis, Empaveli, pegcetacoplan, US, FDA, Approval,  Paroxysmal Nocturnal Hemoglobinuria

• The approval is based on P-III head-to-head PEGASUS study assessing Empaveli vs Soliris (eculizumab) in 80 adults with PNH. Participants should be Soliris stable for at least 3mos. with a Hgb level of <10.5 g/dL at the screening visit
• Results: The study met its 1EPs demonstrating superiority to Soliris for the change from baseline in hemoglobin level @16wks. with an adjusted mean increase of 3.84 g/dL of Hgb. Additionally, therapy met non-inferiority on the endpoint of transfusion avoidance (85% vs 15%)
• Empaveli is the first and only targeted C3 therapy, approved in the US for use in adults with PNH who are treatment naïve and patients switching from the C5 inhibitors Soliris & Ultomiris

Janssen's Rybrevant (amivantamab-vmjw) Receives the US FDA's Approval as the First Targeted Treatment for Patients with NSCLC with EGFR Exon 20 Insertion Mutations

Published: May 24, 2021 | Tags: Janssen, Rybrevant, amivantamab-vmjw, US, FDA, Approval, NSCLC, EGFR Exon 20 Insertion Mutations

• The US FDA has granted accelerated approval which is based on P-I CHRYSALIS study assessing Rybrevant as a monothx. in patients metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations whose disease has progressed on or after platinum-based CT
• The FDA has also approved Guardant Health's Guardant360 CDx liquid biopsy blood test as a CDx for use with Rybrevant
• The approval follows the FDA's BTD in Mar'2020 and to initiate PR of the BLA in Dec'2020. Rybrevant is a fully human bispecific antibody directed against EGFR and MET receptors

Lantheus Pylarify (piflufolastat F 18) Injection Receives the US FDA's Approval for the Treatment of Prostate Cancer

Published: May 27, 2021 | Tags: Lantheus, Pylarify, piflufolastat F 18, Injection, US, FDA, Approval, Prostate Cancer

• The approval is based on data from 2 pivotal studies i.e OSPREY & CONDOR evaluating the safety & diagnostic performance of Pylarify in patients with prostate cancer
• Results: 1st study showed improvement in specificity & PPV compared with conventional imaging for metastatic prostate cancer & 2nd study showed high CLRs & detection rates includes low PSA values (median PSA is 0.8 ng/mL) for biochemically recurrent prostate cancer & non-informative baseline imaging
• Pylarify is the 1st commercially available approved PSMA PET imaging agent for prostate cancer & will be available immediately in mid-Atlantic & southern regions, expected to be broadly available in the US at the end of 2021

Amgen's Lumakras (sotorasib) Receives the US FDA's Approval as the First KRAS Blocking Cancer Therapy

Published:May 31, 2021 | Tags: Amgen, Lumakras, sotorasib, US, FDA, Approval, First KRAS Blocking Cancer Therapy

• The approval is based on the CodeBreaK 100 trial evaluating the efficacy & tolerability of Lumakras (960mg, qd) in 124 patients with KRAS G12C mutation-positive NSCLC whose disease had progressed after prior treatment with immunotherapy or CT
• Results: ORR (36%); DCR (81%); mDoR (10 mos.); durable response with a positive benefit-risk profile, 9% showed permanent discontinuation due to AEs
• The company is conducting biomarker testing & a patient support program for NSCLC. The therapy is also being studied in multiple other solid tumors

BridgeBio and Helsinn's Truseltiq (infigratinib) Receive the US FDA's Approval for Cholangiocarcinoma

Published: May 31, 2021 | Tags: BridgeBio, Helsinn, Truseltiq, infigratinib, US, FDA, Approval, Cholangiocarcinoma

• The approval is based on a P-II study assessing Truseltiq (125mg, qd, for 21 days of 28-day cycles) in 108 patients who had undergone at least one prior treatment for advanced CCA. Out of 108, 107 had Stage IV CCA
• Results: ORR (23%); mDOR (5mos.); presented at ASCO 2021. Additional marketing applications for Truseltiq are currently under review in Australia and Canada under Project Orbis
• Truseltiq is an ATP-competitive, TKI of FGFR. BridgeBio, through its affiliate QED & Helsinn will co-commercialize Truseltiq in the US under its collaboration signed in Mar'2021

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