• The US FDA approved 1 NDAs and 6 BLA in November 2022, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 73 novel products in 2022
• In November 2022, the major highlights drugs were Imfinzi (durvalumab) + Imjudo (tremelimumab) approval for metastatic non-small cell lung cancer, Elahere for platinum-resistant ovarian cancer
• PharmaShots has compiled a list of a total of 7 new drugs approved by the US FDA in November 2022

1. Regeneron’s Libtayo (cemiplimab-rwlc) Receives the US FDA’s Approval as 1L Treatment for Advanced Non-small Cell Lung Cancer

Libtayo

Active ingredient: cemiplimab                                                  Approved: November 09, 2022

Company: Regeneron                                                                Disease: Non-Small Cell Lung Cancer

• The approval was based on the P-III (EMPOWER-Lung 3) trial evaluating Libtayo (350mg, IV, q3w) + Pt-doublet CT vs CT alone in a ratio (2:1) in 466 patients with LA or metastatic NSCLC irrespective of PD-L1 expression or tumor histology with no ALK, EGFR or ROS1 aberrations
• The results showed an improvement in OS, m-OS & m-PFS (22 vs 13mos.) & (8 vs 5mos.) with 29% & 44% relative reduction in risk of death & disease progression; 12mos. probability of survival (66% vs 56% & 38% vs 16%), no new Libtayo safety signals were reported
• The therapy showed an ORR (43% vs 23%), m-DoR (16 vs 7mos.), serious AEs were reported in 25% & AEs also lead to treatment discontinuations in 5%

2. Seagen’s Adcetris (brentuximab vedotin) Receives the US FDA’s Approval for High-Risk Hodgkin Lymphoma

Adcetris

Active ingredient: brentuximab vedotin                                  Approved: November 10, 2022

Company: Seagen                                                                     Disease: Hodgkin Lymphoma

• The US FDA has approved Adcetris for high-risk cHL in combination with doxorubicin, vincristine, etoposide, prednisone, and cyclophosphamide for pediatric patients aged ≥2yrs.
• The approval was based on the P-III (NCI)-sponsored study (AHOD1331), conducted by the Children's Oncology Group (COG) & funded by NCI evaluating Adcetris + CT (AVE-PC) vs CT (ABVE-PC) in 587 patients aged 2-21yrs. across 151 institutions. The results showed a superior EFS over ABVE-PC & 59% reduction in risk of disease progression or relapse, second cancer, or death
• Seagen & Takeda collaborated to develop Adcetris where Seagen has the US & Canadian commercialization rights, and Takeda has rights to commercialize the product globally

3. AstraZeneca’s Imfinzi (durvalumab) + Imjudo (tremelimumab) Receive the US FDA’s Approval for Metastatic Non-Small Cell Lung Cancer

Imfinzi 

Active ingredient: durvalumab                                                 Approved: November 14, 2022

Company: AstraZeneca                                                             Disease: Non-Small Cell Lung Cancer

• The approval was based on the P-III (POSEIDON) trial evaluating Imfinzi + Imjudo and CT vs CT alone in 1013 patients with metastatic NSCLC
• The results showed that the patients treated with the combination therapy achieved a 23% reduction in risk of death, patients were alive @2yrs. (33% vs 22%), 28% reduction in risk of disease progression or death, improvement in OS (25%), patients were alive @3yrs. (25% vs 13.6%). The safety profile was consistent with the known profiles of each medicine with no new safety signals. The results were presented at ESMO 2022 & published in the Journal of Clinical Oncology
• Based on the (POSEIDON) trial results, the regulatory application is currently under review in the EU, Japan & multiple other countries

4. ImmunoGen’s Elahere Receive the US FDA’s Approval for the Treatment of Platinum-Resistant Ovarian Cancer

Elahere

Active ingredient: mirvetuximab soravtansine                         Approved: November 14, 2022

Company: ImmunoGen                                                               Disease: Ovarian Cancer

• The approval was based on the P-III (SORAYA) trial evaluating Elahere in 106 patients with Pt-resistant ovarian cancer whose tumors expressed high levels of FRα & treated with one to three prior systemic treatment regimens & required to have received Avastin
• The results showed an ORR (31.7%) by the investigator incl. CR rate (4.8%) & PR rate (26.9%), m-DoR was 6.9mos., DCR (51.4%), 71.4% reduction in tumor size. The safety has been evaluated in a pooled analysis across 3 studies
• The therapy is also being evaluated in the (MIRASOL) trial with expected results in early 2023. The US FDA has also approved VENTANA FOLR1 (FOLR1-2.1) RxDx assay for use as a CDx device to identify patients eligible for Elahere

5. Sun Pharmaceutical’ Sezaby Receives the US FDA’s Approval for the Treatment of Neonatal Seizures

Sezaby

Active ingredient: phenobarbital                                                   Approved: November 18, 2022

Company: Sun Pharmaceutical                                                     Disease: Seizures

• The US FDA has approved Sezaby (phenobarbital sodium powder for injection) for the treatment of neonatal seizures with expected availability in the US in Q4’23
• The approval was based on the results from the P-II (NEOLEV2) study evaluating levetiracetam vs phenobarbital in 94 neonates with neonatal seizures. Patients treated with levetiracetam were seizure-free (73% vs 25%) after 24hrs.
• SEZABY is a benzyl alcohol-free and propylene glycol-free formulation of phenobarbital sodium powder for injection & marks the first product for neonatal seizures in term and preterm infants in the US. The therapy received ODD from the US FDA for the same indication

6. Provention Bio's TZIELD (teplizumab-mzwv) Receives the US FDA's Approval of BLA for the Treatment of Stage 2 Type 1 Diabetes

TZIELD

Active ingredient: teplizumab-mzwv                                               Approved: November 21, 2022

Company: Provention Bio                                                                Disease: Type 1 Diabetes

• The US FDA has approved the BLA for TZIELD (anti-CD3-directed Ab) in adult and pediatric patients aged ≥8yrs. with stage 2 T1D. TZIELD inj. is provided as a sterile, preservative-free, clear & colorless solution in a 2mg/2mL (1mg/mL) single-dose vial for IV use
• Under the terms of a co-promotion agreement with Sanofi in Oct 2022, the companies collaborated to launch TZIELD in the US for the delay in the onset of clinical T1D in at-risk individuals
• The company has launched a patient support program i.e., COMPASS that provides access to patients to use TZIELD & also offers financial assistance options i.e., copay assistance for out-of-pocket costs to eligible patients

7. uniQure’s Hemgenix (etranacogene dezaparvovec-drlb) Receives the US FDA’s Approval for the Treatment of Hemophilia B

Hemgenix

Active ingredient: etranacogene dezaparvovec                        Approved: November 23, 2022

Company: uniQure                                                                       Disease: Hemophilia B

• The US FDA has approved Hemgenix (etranacogene dezaparvovec-drlb) in adults aged ≥18yrs. with hemophilia B
• The approval was based on the P-III (HOPE-B) trial evaluating Hemgenix (2x10¹³gc/kg, IV) in 54 patients which showed a reduction in the rate of annual bleeds & 94% of patients discontinued factor IX prophylaxis and remained prophylaxis-free with mean factor IX activity of 39% @6mos. and 36.7% @24mos. post-infusion, 54% reduction in ABR bleeds over 6mos. on factor IX prophylactic replacement therapy
• Under the terms of the agreement, CSL gets an exclusive right globally to Hemgenix in May 2021 & responsible for the development, registration & commercialization of the therapy

Related Post: Insights+: The US FDA New Drug Approvals in October 2022