Insights+: The US FDA New Drug Approvals in December 2020

The US FDA has approved 7 NDAs and 2 BLA in Dec 2020, 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 105 novel products so far in 2020, including 9 in Dec 2020.  

Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 9 new drugs approved by the US FDA in Dec 2020. 

The US FDA Approved Gallium 68 PSMA-11 as the First PSMA-Targeted PET Imaging Drug for Men with Prostate Cancer 

Published: Dec 01, 2020 | Tags: US, FDA, Approves, Gallium 68 PSMA-11, PET imaging, PSMA Positive Lesions, Men, Prostate Cancer 

• The approval is based on two prospective studies assessing Ga 68 PSMA-11 in 960 men with prostate cancer. The first trial enrolled 325 patients & the second trial enrolled 635 patients with biopsy-proven prostate cancer who underwent PET/CT or PET/MRI scans and who had rising serum PSA levels after initial prostate surgery or radiotherapy  
• 74% of patients had at least one positive lesion detected by Ga 68 PSMA-11 PET in at least one body region  
• Ga 68 PSMA-11 is a radioactive diagnostic agent that is administered in the form of an IV injection. The approval is granted to the University of California  

Vanda’s Hetlioz (tasimelteon) Received the US FDA’s Approval for Nighttime Sleep Disturbances in Smith-Magenis Syndrome  

Published: Dec 01, 2020 | Tags: Vanda, Hetlioz, tasimelteon, US, FDA, Approval, Treatment, Nighttime Sleep Disturbances, Smith-Magenis Syndrome 

• The approval is based on study assessing Heltioz vs PBO in both adults with SMS taking the capsule formulation and children with SMS taking the liquid formulation. The safety profile of the therapy is in consistent with previous study conducted for the treatment of Non-24-Hour Sleep-Wake Disorder 
• Hetlioz capsules will available imminently for adults and Hetlioz LQ liquid formulation for children is expected to be available in Q1’21  
• Hetlioz is melatonin receptor agonist & is an approved therapy in the US & EU 

Roche’s Gavreto (pralsetinib) Received the US FDA’s Approval for Advanced or Metastatic RET-Mutant and RET Fusion-Positive Thyroid Cancers 

Published: Dec 02, 2020 | Tags:  Roche’s, Gavreto, (pralsetinib), Receives, US FDA’s Approval, Advanced or Metastatic RET-Mutant, RET Fusion-Positive Thyroid Cancers 

• The approval is based on P-I/II ARROW study involve the assessing Gavreto (400mg, qd) n people with rearranged during transfection (RET) fusion-positive NSCLC, RET-mutant MTC, RET fusion-positive thyroid cancer and other RET-altered solid tumors 
• The study demonstrated durable clinical activity in people with/out prior therapy and regardless of RET alteration genotypes 
• Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations  

Biocryst’s Orladeyo (berotralstat) Received the US FDA’s Approval to Prevent Attacks of Hereditary Angioedema (HAE) 

Published: Dec 03, 2020 | Tags: Biocryst, Orladeyo, (berotralstat), Receives, US, FDA, Approval, hereditary angioedema (HAE) 

• The approval is based on a pivotal P-III APeX-2 trial assessing Orladeyo (150 mg, qd) in adults and pediatric patients aged ≥12yrs. with HAE 
• The study demonstrated reduction attacks @24wks. while the reduction was sustained through 48wks. with mean HAE attack rates of 2.9 attacks/mos. In the long-term open-label APeX-S trial, patients completing 48wks. of therapy had a mean attack rate of 0.8 attacks/mos. 
• Orladeyo (PO) is designed specifically to prevent attacks of HAE in adults and pediatric patients aged ≥12yrs 

Athenex’s Klisyri (tirbanibulin) Received the US FDA’s Approval for Actinic Keratosis on the Face or Scalp 

Published: Dec 15, 2020 | Tags: Athenex, Klisyri, (tirbanibulin), Receives, US, FDA, Approval, Actinic Keratosis, Face, Scalp 

• The approval is based on P-III studies KX01-AK-003 and KX01-AK-004 that evaluated the efficacy and safety of Klisyri (tirbanibulin, ointment 1%, 10 mg/g) vs vehicle in 702 adult patients in the ratio of (1:1) with actinic keratosis of the face or scalp  
• Both the studies achieved their 1EPs defined as 100% clearance of the AK lesions @57 days within the treatment areas, complete clearance (44% vs 5% and 54% vs 13%) respectively  
• Klisyri is the first FDA approved product for Athenex and will be launched in partnership with Almirall in the US during Q1’21  

Myovant’s Orgovyx (relugolix) Received the US FDA’s Approval as the First Oral GnRH Receptor Antagonist for Advanced Prostate Cancer 

Published: Dec 15, 2020 | Tags: Myovant, Orgovyx, (relugolix), Advanced Prostate Cancer 

• The approval is based on a P-III HERO study assessing Relugolix (360mg loading dose followed by 120mg, qd) vs leuprolide acetate (3mos. depot injection) in patients with advanced prostate cancer. The therapy is expected to be available in Jan’2021 
• Results: met its 1EPs, sustained testosterone suppression to castrate levels (< 50 ng/dL) @48wks. (96.7% vs 88.8%).  
• 2EPs include @day4 & 15 suppression of testosterone to castrate levels (56% & 99% vs 0% & 12%); profound suppression of testosterone (< 20 ng/dL) @day15 (78% vs 1%); reduction in PSA by 65% @day15 and by 83% @day 29 

MacroGenics’ Margenza (margetuximab-cmkb) Received the US FDA’s Patients with Pretreated Metastatic HER2-Positive Breast Cancer 

Published: Dec 16, 2020 | Tags: MacroGenics, Margenza, Receives, US, FDA, Metastatic, HER2-Positive Breast Cancer 

• The approval is based on P-III SOPHIA trial involves assessing of Margenza (IV, 15 mg/kg, q3w) vs Herceptin (trastuzumab, IV, 6mg/kg or 8mg/kg for loading dose) both combined with CT in 536 patients in a ratio (1:1) with HER2-positive m-BC who have received two or more prior anti-HER2 regimens, at least one of which was for metastatic disease.  
• The study showed 24% reduction in the risk of disease progression or death, ORR (22% vs 16%), The final OS analysis is expected in the H2’21  
• Margenza is an Fc-engineered, mAb that targets the HER2 oncoprotein, also being evaluated in multiple trials for HER2+ gastroesophageal cancer & various HER2+ tumors 

Ridgeback’s Ebanga (mAb114) Received the US FDA’s Approval for the Treatment of Ebola 

Published: Dec 22, 2020 | Tags: Ridgeback, Ebanga, mAb114, Receives, US, FDA, Approval, Ebola 

• Ebanga is now approved for treatment of infection caused by Zaire ebolavirus in adult & pediatric patients (including neonates born to a mother who is RT-PCR+ for Zaire ebolavirus infection) 
• The efforts of the PALM study demonstrated Ebanga’s safety and efficacy in a randomized controlled trial conducted during the 2nd largest and longest outbreak in DRC history 
• In 2020, Ridgeback initiated a compassionate use protocol for Ebola patients during the DRC’s 11th Ebola outbreak in Équateur Province and will provide it to all PCR+ Ebola patients under the protocol 

Urovant’s Gemtesa (vibegron) Received the US FDA’s Approval for Overactive Bladder  

Published: Dec 23, 2020 | Tags: Urovant, Gemtesa, Vibegron, US, FDA, Approval, Treatment, Overactive Bladder, OAB 

• The approval is based on P-III EMPOWUR and EMPOWUR long term extension study that involves assessing Gemtesa (75mg, qd) vs PBO in ~4000 patients with OAB for 12wks. 
• The study resulted in reductions in daily UUI, micturitions, and urgency episodes and an increase in the volume voided 
• Gemtesa is a small-molecule β3 adrenergic receptor agonist therapy that relaxes the detrusor bladder muscle so that the bladder can hold more urine, thus reducing symptoms of OAB. Gemtesa is the first oral branded FDA’s approved OAB medication since 2012, and it is the first product approval for Urovant Sciences 

Related Post: Insights+: The US FDA New Drug Approvals in November 2020