More than 10 new Q4 drugs are expected to be approved for sale in 2020 involving Sanofi, Pfizer and BMS

A number of new products are expected to be regulatoryly approved to enter the market by the end of the year, including new drugs from Sanofi, Pfizer and Pershing, which will open up new areas of commerce or shake up their treatments when they go on sale, according to a recent article by Xconomy, a leading U.S. health information website.
following is the article's list of the 12 most noteworthy regulatory approvals for the fourth quarter of 2020.
One of the drugs, Inmazeb, which was approved by the FDA on October 15th, is a three-anti-cocktail therapy developed by Regenerative, the first Ebola treatment drug approved in the United States for the treatment of infections caused by the Zairian Ebola virus, which can be used in adults and children infected, including newborns of infected mothers who have tested positive.
Inmazeb was developed by Regeneratives using its proprietary VelociSuite® rapid response technology, which is currently being used to develop a new dual-anti-cocktail therapy, REGN-COV2, for new coronavirus pneumonia (COVID-19).
the remaining 11 drugs are as follows: 1. SPN-812 Adaptation: Attention Deficit ADHD PDUFA Date: November 8 - The first review of SPN-812 was developed by Supernus Pharmaceuticals, a new non-irritating treatment for ADHD in children and adolescents.
5-serotonin-dephetamine regulator (SNMA) inhibits the reuptake of dethyroidine in transporters and has been approved in Europe for years to treat depression.
FDA approval, SPN-812 would be the first new treatment for ADHD in a decade.
nearly 6.1 million children and adolescents with ADHD in the United States, there is an urgent need for an unregulated treatment that is different from the mechanisms in which existing therapies work.
SPN-812 New Drug Application (NDA) is based on data from a clinical development program that includes four Phase III trials that looked at patient populations of children ages 6-17.
4 key clinical trials showed that the overall score of ADHD-RS-5 declined as early as week 1 and continued until the end of clinical studies, while the subsoometers of ADHD/impulsivity and inattulation improved.
SPN-812 has acceptable safety, the rate of adverse events is low, and the rate of drug suspension is low.
the FDA has set a target date for action under the Prescription Drug User Charges Act (PDUFA) as November 8, 2020.
the FDA's decision is expected by Friday because it happens to be a weekend.
2. Sutimlimab (BIVV009) Adaptation: Condensation Anaemia (Autoimmune Hemolytic Anemia) PDUFA Date: November 13 - First review sutimlimab developed by Sanofi to treat hemolytic biological products (BLA) in adult patients with primary condensate disease (CAD) is under priority review by the FDA.
sutimlimab is a monoclonal antibody that targets the underlying causes of CAD hemolysis by selectively suppressing complement C1s in the classical complement pathway of the immune system.
if approved, sutimlimab would be the first and only drug to treat CAD hemolysis.
, the incidence of CAD is about 16 parts per million, with an estimated 12,000 cases in the United States, Europe and Japan, and about 5,000 cases of CAD in the United States alone.
previously, Sutimlimab had been granted the orphan drug qualification and breakthrough drug qualification for CAD treatment.
the submission of the sutimlimab BLA, based on data from an open-label single-arm critical Phase III CARDINAL clinical trial conducted in primary CAD patients.
in this trial, sutimlimab reached the main endpoint of compound efficacy: 54% of patients had increased hemoglobin levels over baseline ≥2g/dL, 62% normalized hemoglobin levels at week 26 (≥12g/dL), and 71% did not have blood transfusions during weeks 5-26.
In addition, trials have shown that sutimlimab has reached a secondary end point: showing improvements in key indicators of the disease process, including improvements in hemoglobin, normalization of bilium, and improvements in the evaluation of chronic disease treatment function assessment (FACIT) fatigue scores.
3. Margetuximab Adaptation: Breast Cancer PDUFA Date: December 18 - First review margetuximab developed by MarcroGenics, an Immune-enhanced monoimmune optimized Fc domain optimized for targeting human skin growth factor recipient 2 (HER2), has been awarded fast-track qualification by the FDA for the treatment of metastasis or late-stage local HER2-positive breast cancer patients who have previously received anti-target 2 targeted therapies.
If approved, margetuximab would provide an alternative treatment for her2-positive metastasis breast cancer patients, although the drug could face fierce competition from two recently approved breast cancer drugs, including AstraZeneca's antibody conceding drug Enhertu and Seattle Genetics' target drug Tukysa, renamed Seagen.
margetuximab BLA is supported by the results of the Key III SOPHIA study.
the study obtained the main endpoint: margetuximab plus chemotherapy significantly extended the progression-free lifetime (medium PFS: 5.8 months vs. 4.9 months; HR=0.76) compared to curvature monoantigen (a HER2 targeted monoantigen) and chemotherapy, and had a commoable safety and tolerance.
biomedtracker analysts said that while Margetuximab had not significantly improved total lifetime (OS) in its second interim analysis, the trend in OS in the intended treatment population was encouraging, particularly in the CD16A 158F allied population.
previously released data show that the PFS advantage over the crayto-bead monoantigen will be sufficient to support the approval of the BLA used in combination with chemotherapy.
4. lisocabtagene maraleucel (liso-cel) Adaptation: diffuse Large B-Cell Lymphoma (DLBCL) PDUFA Date: 16 November - First review of liso-c Developed by BMS and likely to receive FDA approval in mid-November, the drug is expected to go on sale under the trade name Breyanzi, which will compete with Gilead Sciences' Yescarta and Novaral's Kymriah.
liso-cel is an autobiographic, CD19-guided, chisellular antigen (CAR) T-cell therapy consisting of purified CD8-plus and CD4-T cells in a specific proportion (1:1).
-cel was acquired by BMS in 2019 through a $74 billion acquisition of New Base and is currently under regulatory review in the United States, the European Union and Japan for third- or multi-line therapy recurrence or resuscable DLBCL.
BMS's U.S. review was delayed because it submitted additional information to the FDA, which was considered a major change to the BLA, with the new PDUFA target date of November 16.
liso-cel is different from its competitors in that purified CD4-plus and CD8-plus cells are prepared separately and then infused in the same target dose order.
this complicates production, it is considered to reduce product variability and improve safety, allowing use in outpatient clinics.
liso-cel BLA, supported by TRANSCEND clinical trial data.
the trial was conducted in 269 patients with relapsed/re treatable large B-cell lymphoma (including DLBCL), with a total remission rate (ORR) of 73% and a total remission rate (CR) of 53% in patients with assessable efficacy.
results were better than those from yescarta's ZUMA-1 trial and Kymriah's JULIET trial, which had a CR rate of 51% and 32%, respectively.
study, only 6 (2%) patients treated with liso-cel developed 3/4 cytokine release syndrome (CRS), while Yescarta and Kymriah had 13% and 23%, respectively.
5. Eysuvis Allergy: Dry Eye PDUFA Date: October 30 - The second review Eysuvis was developed by Kala Pharmaceuticals, a solution for the eye of loteprednol etabonate for short-term treatment of symptoms and signs of dry eye disease, but received a full FDA response letter (CRL) in August 2019.
, Eysuvis is positioned as a short-term treatment for dry eyes rather than maintenance.
because Both Novarma's Lifitegrast and Eljian's Restasis (the emulsion for cyclosporine eye) are used as long-term treatments, this will make Eysuvis uniquely differentiated in this competitive adaptive range.
CRL noted that additional clinical trial efficacy data were needed to support resubmit, which led to Phase 3 trials to complement previously completed Phase 2 trials and the first 2 Phase 3 Efficacy and Safety Trials (STRIDE 1, STRIDE 2).
Kala resubmitted its application and STRIDE 3 trial data in April, with the PDUFA target date for action on October 30.
study, which met two major symptom endpoints, showed significant improvements in the severity of eye discomfort in the overall ITT population and in the pre-designated ITT patient subgroup.
6. Zokinvy Adaptation: Premature Aging PDUFA Date: November 20 - The first review of Zokinvy, developed by Eiger, is currently under FDA priority review for the treatment of premature aging (Progeria, also known as Hudkinson-Gilford premature aging syndrome, HGPS) and premature aging-like nuclear fibrinosis (Progeroid Lampathies).
approved, Zokinvy would be the world's first drug to treat premature aging.
last December, Eiger launched the Zokinvy NDA rolling submission, which was completed in March this year.
, Zokinvy was awarded the FDA's Orphan Drug Eligibility (ODD), Breakthrough Drug Qualification (BTD) and Rare Pediatric Disease Qualification (RPDD) for the treatment of premature aging and early aging-like nuclear fibrinology.
results from the ProLon1 and ProLon2 trials, published in the April 2018 issue of the Journal of the American Medical Association (JAMA), assessed the effects of Zokinvy's treatment on mortality compared to untreated patients over the same period.
data from two studies showed that taking Zokinvy reduced the risk of death by 77% after 2.2 years of follow-up (HR-0.23; 95% CI: 0.06-0.90; p-0.04).
Lumasiran Adaptation: PDUFA Date: December 3 - First review lumasiran developed by Alnylam and currently under FDA priority review.
If approved, the drug would be the first treatment for the ultra-rare pathogenic hyperchloric acid uremia type 1 (PH1) and the third approved RNAi treatment following the pioneering success of the company's drug patisiran in 2018.
PH1 is characterized by excessive production of heralic acid in the liver, which leads to build-up in the kidneys, a gradual decline in kidney function, and usually develops into renal failure.
lumasiran is a subsurfic injection RNAi drug that targets hydroxy acid oxidase 1 (HAO1), hao1 encodes ethanolate oxidase (GO).
lumasiran inhibits and normalizes the production and normalization of oxalic acid (a metabolite directly involved in PH1 pathophysiology) in the liver by silencing HAO1 and consuming GO enzymes, potentially blocking the progression of PH1 disease.
lumasiran is the first treatment to show a significant reduction in urinary acid excretion.
results from Phase III ILLUMINATE-A show that lumasiran therapy significantly reduced uric acid levels by 65% compared to baseline levels and by 54% compared to placebos.
addition, 52 percent of patients in the lumasiran treatment group had uric acid levels in the normal range, 84 percent were near normal, and those in the placebo group were not at or near normal levels.
although no clinical benefits were observed due to the shortness of the trial, the significant reduction in herbic acid is expected to have a beneficial effect on renal function and disease progress as follow-up time increases.
8. Danyelza Adaptation: Neuroendocrine Tumor PDUFA Date: November 30 - The first review of Danyelza (naxitamab) was developed by Y-mAbs Therapeutics using its proprietary MULIT TAG protein platform, a humanized 3F8 monoclonal antibody targeting GD2 antigens.
November, the FDA accepted a rolling BLA application for Danyelza's treatment of relapsed/recurring high-risk neuroblastoma.
if approved, Danyelza will become Roche Avastin since 2009