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

The US FDA has approved 7 NDAs and 1 BLA in Feb 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 12 novel products in 2021.

Additionally, last year in 2020, the US FDA has approved 114 novel products. PharmaShots has compiled a list of a total of 8 new drugs approved by the US FDA in Feb 2021

TG Therapeutics Ukoniq (umbralisib) Received the US FDA's Approval for R/R Marginal Zone Lymphoma and Follicular Lymphoma

Published: Feb 02, 2021 | Tags: TG Therapeutics, Ukoniq (umbralisib), Receives, US, FDA, Approval, Treatment, R/R, Marginal Zone Lymphoma, Follicular Lymphoma

The approval is based on P-II UNITY-NHL trial involves assessing Ukoniq (800 mg, qd) in 69 patients with MZL who has received at least 1 prior anti-CD20 based regimen and in 117 patients with FL who received at least 2 prior systemic therapies
Results: ORR (49% vs 50%) as assessed by IRC, CR (16% vs 3.4%), PR (33% vs 39%), DoR of FL was 11.1 mos.
Ukoniq is the 1st oral inhibitor of PI3K delta and CK1 epsilon. Additionally, it has received BTD for MZL and ODD for MZL and FL

Durect's Posimir (bupivacaine solution) Received the US FDA's Approval for the Treatment of Post-Surgical Pain

Published: Feb 02, 2021 | Tags: Durect, Posimir (bupivacaine solution), Receives, US, FDA, Approval, Treatment, Post-Surgical Pain

The approval is based on clinical trial involves assessing Posimir (4mg) vs PBO (12mg) in patients in patients undergoing arthroscopic subacromial decompression surgery with an intact rotator cuff for the first 72hrs.
The results demonstrated an improvement in both primary outcome measures i.e. 20% reduction in mean pain intensity on a 0-10 point pain scale; 67% reduction in IV morphine-equivalent rescue opioid use
Posimir is the only approved sustained-release bupivacaine product indicated for up to 72hours of post-surgical analgesia from a single application

Merck KGaA's Tepmetko (tepotinib) Received the US FDA's Approval for Patients with Metastatic NSCLC with METex14 Skipping Alterations

Published: Feb 04, 2021 | Tags: Merck KGaA, Tepmetko (tepotinib), Receives, US, FDA, Approval, Patients, Metastatic NSCLC, METex14 Skipping Alterations

The approval is based on pivotal P- II VISION study that involves assessing of Tepmetko (450mg, qd) as monothx. in 152 patients with a median age of 73 years with advanced/ m-NSCLC with METex14 skipping alterations
Results: treatment-naïve patients & previously treated patients, ORR (43% & 43%); mDoR (0.8 and 11.1mos.); DoR @6mos. (67% & 75%); DoR @9mos. (30% & 50%) respectively
Tepmetko is an oral MET inhibitor that inhibits the oncogenic MET receptor signaling caused by MET (gene) alterations and approved for both treatment naïve and previously treated METex14 positive NSCLC patients

Regeneron's Evkeeza (evinacumab-dgnb) Received the US FDA's Approval for Patients with Homozygous Familial Hypercholesterolemia

Published: Feb 12, 2021 | Tags: Regeneron, Evkeeza (evinacumab-dgnb), Receives, US, FDA, Approval, Patients, Homozygous Familial Hypercholesterolemia

The approval is based on the P-III ELIPSE trial that involves assessing Evkeeza (15 mg/kg, IV, q4w) + vs PBO in 65 patients aged =12yrs. with HoFH for 24wks.
The study met 1EPs i.e. 49% reduction in LDL-C (47% vs 2%), patients also experienced a reduction in the level of ApoB, non-HDL-C. Reductions in LDL-C were observed as early as weeks 2 and maintained throughout week 24 and the open-label trial period (through week 48).
Evinacumab is a mAb targeting ANGPTL3 and is under FDA's PR following the BTD granted in 2017

G1 Therapeutics Cosela (trilaciclib) Received US FDA's Approval as the First Myeloprotection Therapy for Extensive-Stage Small Cell Lung Cancer

Published: Feb 15, 2021 | Tags: G1 Therapeutics, Cosela (trilaciclib), Receives, US, FDA, Approval, First, Myeloprotection Therapy, Extensive-Stage Small Cell Lung Cancer

The approval is based on three clinical trials involves assessing Trilaciclib +carboplatin/etoposide (+/- atezolizumab) and topotecan CT regimens in patients with ES-SCLC. 90% of all patients with ES-SCLC will receive at least one of these regimens
The result show reductions in the incidence and duration of severe neutropenia, positive impact on RBC transfusions and other myeloprotective measures, and impacted anemia and the need for rescue interventions such as growth factors
Trilaciclib is the 1st therapy designed to help protect bone marrow during CT and has received the US FDA's BTD in 2019 and expected to be commercially available in Mar'2021

Sarepta's Amondys 45 (casimersen) Received the US FDA's Approval for DMD in Patients Amenable to Skipping Exon 4

Published: Feb 25, 2021 | Tags: Sarepta Therapeutics, Amondys 45 (casimersen), Receives, US, FDA, Approval, Treatment, Duchenne Muscular Dystrophy (DMD)

The approval is based on ESSENCE trial assessing Amondys 45 vs PBO in patients with DMD with a confirmed mutation amenable to exon 45 skipping. The study is expected to conclude in 2024
The study resulted in a significant increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45. Consistent with the accelerated approval, the continued approval of Amondys 45 may contingent on confirmation of clinical benefit in a confirmatory trial.
Amondys 45 is the third RNA exon-skipping treatment for DMD approved in the US. The company will initiate the commercial distribution of therapy in the US imminently

B. Braun's Acetaminophen Injection Received the US FDA Approval in Multiple Doses

Published: Feb 25, 2021 | Tags: B. Braun, Acetaminophen Injection, Receives, US, FDA, Approval, Multiple Doses

The US FDA has approved the acetaminophen injection available in multiple doses. The company has used dual-sourced API to reduce potential supply risks
Acetaminophen injection is available in flexible PAB IV bags in both 1,000 mg in 100 mL and 500 mg in 50 mL doses. PAB IV bags are not made with PVC, DEHP, or latex
Acetaminophen injection is the first multiple injectable drugs. The company plans to launch it in the coming years to meet the needs of patients for medications in high demand

BridgeBio and Origin's Nulibry (fosdenopterin) Received the US FDA's Approval as the First Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A

Published: Feb 28, 2021 | Tags: BridgeBio and Origin, Nulibry (fosdenopterin), Receives, US, FDA, Approval, First, Therapy, Reduce, Risk, Mortality, Patients, MoCD Type A

The FDA has approved Nulibry based on three clinical trials compared to data from a natural history study in patients with MoCD Type A
The results from three clinical trials showed improvement in overall survival and an 82% reduction in the risk of death, @3yrs. probability of survival in rcPMP-treated patients (84% vs 55%), reduction of urine concentrations of SSC and reduction was sustained with long-term treatment over 48mos., the high infant mortality rate
Nulibry is a first-in-class cPMP substrate replacement therapy and reviewed under PR and has received FDA's ODD, BTD and RPDD. With this approval, the FDA also issued an RPD PRV to Origin