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

The US FDA has approved 5 NDAs and 1 BLA in Nov 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 96 novel products so far in 2020, including 6 in Nov 2020. Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 6 new drugs approved by the US FDA in Nov 2020.

Sedor's Sesquient Received the US FDA's Approval for Status Epilepticus

Published: Nov 09, 2020 | Tags: Sedor Pharmaceuticals, US FDA approval, Sesquient, Treatment, Status Epilepticus, Adult and Pediatric patients

The US FDA has approved Sesquient (fosphenytoin sodium for injection) for the treatment of status epilepticus in adult and pediatric patients
Sedor is actively in discussions to license the rights to Sesquient for NA, EU, and other territories, except for the People's Republic of China, where it has already been licensed
Sesquient is the first and only FDA-approved room temperature stable fosphenytoin and can help HCPs to treat status epilepticus patients and reduce hospital costs associated with this condition

Eiger's Zokinvy (lonafarnib) Received the US FDA's Approval for Treatment of Progeria and Processing-Deficient PL

Published: Nov 20, 2020 | Tags: approval, Deficient, Eiger, lonafarnib, PL, Processing, Progeria, receives, Treatment Us FDA, Zokinvy

The Progeria Research Foundation and Eiger reported the US FDA's approval of Zokinvy (lonafarnib) for the treatment of HGPS or progeria and processing-deficient progeroid laminopathies
Zokinvy reduced the incidence of mortality by 60% & increased the average survival time by 2.5 yrs. Additionally, the FDA has issued an RPD priority review voucher to Eiger
Eiger plans to sell the PRV and will share the proceeds equally with PRF, under its supply & collaboration agreement. Zokinvy is a farnesyltransferase inhibitor that has shown a survival benefit in children with Progeria

Alnylam's Oxlumo (lumasiran) Received the US FDA's Approval for Treatment of Primary Hyperoxaluria Type 1 in Pediatric and Adult Patients

Published: Nov 23, 2020 | Tags: Adult, Alnylam, approval, Hyperoxaluria, Lumasiran, OXLUMO, patients, Pediatric, Primary, receives, Treatment, Type 1, US FDA

The approval is based on P-III ILLUMINATE-A & -B trials. The studies demonstrating reductions in urinary oxalate and encourage safety and tolerability in pediatric and adult patients
The ILLUMINATE-A showed that Oxlumo met its 1EP i.e. change in 24hrs. (65% vs 12%) compared to PBO, the study also achieved significant results for all 6 tested 2EPs
In ILLUMINATE-B, Oxlumo demonstrated a 72% mean reduction in spot urinary oxalate: creatinine ratio from baseline to 6mos., reduction of oxalate as consistent across all three body wt. categories. Additionally, therapy demonstrated positive results across 2EPs, including additional measures of oxalate

Roche's Xofluza (baloxavir marboxil) Received the US FDA's Approval for Post-Exposure Prevention of Influenza

Published: Nov 24, 2020 | Tags: approval, baloxavir marboxil, Contact, Following, Infected, Influenza, Person, Prevention, receives, Roche, Us FDA, Xofluza

The approval is based on P-III BLOCKSTONE study assessing a single dose of Xofluza vs PBO in household members who were living with someone with influenza confirmed by a rapid influenza diagnostic test
Results: The proportion of household members aged =12yrs. who developed influenza (1% vs 13%), well-tolerated with no new safety signals
Xofluza is the first single-dose influenza medicine approved to prevent influenza for those who have had contact with an infected person. Roche also plans to file sNDA for Xofluza as a treatment for acute uncomplicated influenza in pediatric patients (1-12yrs.) and for the prevention of influenza in the same age group who have been exposed to influenza

Y-mAbs Danyelza (naxitamab-gqgk) Received the US FDA's Approval for Neuroblastoma

Published: Nov 25, 2020 | Tags: (naxitamab-gqgk), approval, Danyelza, Neuroblastoma, receives, US FDA, Y-mAbs Therapeutics

The US FDA has approved Danyelza (40mg/10ml) and is indicated in combination with GM-CSF for pediatric patients aged 1yrs. & older and adult patients with r/r high-risk neuroblastoma in the bone marrow
The indication is approved under accelerated approval regulation based on ORR and DOR. Continued approval for the indication may be contingent upon verification and description of clinical benefits in a confirmatory trial
Danyelza is a mAb that targets the ganglioside GD2 which is highly expressed in various neuroectoderm-derived tumors and sarcomas and has received PR, ODD, BT and RPD from the US FDA

Rhythm's Imcivree (setmelanotide) Received the US FDA's Approval for Chronic Weight Management in Patients with Obesity

Published: Nov 27, 2020 | Tags: Rhythm, Pharmaceutical, IMCIVREE, setmelanotide, Receives, US FDA, Approval, Chronic, Weight, Management, Patients, Obesity

The US FDA has approved Imcivree for chronic weight management in adult & pediatric patients aged =6yrs. with obesity due to POMC, PCSK1 or LEPR deficiency confirmed via genetic testing
The approval is based on results of P-III trials that demonstrated 80% of patients with obesity due to POMC or PCSK1 deficiency achieved >10% weight loss & 45.5% of patients with obesity due to LEPR deficiency achieved >10% weight loss after 1yr of treatment
The US FDA issued a Rare Pediatric Disease PRV to Rhythm with the approval. The PRV can be redeemed to receive PR for any marketing application or sold/ transferred to other companies for their programs. The therapy is under EMA's review for approval