June 15, 2024

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3 new drug applications have been delayed by the FDA

3 new drug applications have been delayed by the FDA: Ublituximab, ONS-5010…


3 new drug marketing applications have been delayed by the FDA: Ublituximab, ONS-5010…


On the last day of May, three new drug applications were delayed by the FDA.


3 new drug applications have been delayed by the FDA: Ublituximab, ONS-5010…




Drug name: Ublituximab

Company Name: TG Therapeutics

Indications: Relapsing Multiple Sclerosis

On May 31, TG announced that the FDA extended the PDUFA date for the Biologics License Application (BLA) for its CD20 monoclonal antibody ublituximab for the treatment of relapsing multiple sclerosis (RMS) to December 28, 2022.

The purpose of the FDA’s extension of the PDUFA date is to allow time to review the information provided by TG in response to the FDA’s request, which the FDA considers to be a significant revision. The submission includes a synthesis and summary of certain clinical information.


Ublituximab is an engineered monoclonal antibody under development.


When ublituximab binds to B cells, it can trigger a series of immune responses including antibody-dependent cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC) to destroy B cells. These cells are thought to be a key contributor to myelin and nerve cell axonal damage.


A unique feature of ublituximab is that it is glycoengineered to remove certain sugar molecules from the antibody, which significantly enhances the potency of ublituximab, especially ADCC activity.


This product BLA submission is based on the results of the ULTIMATE I and II trials, two identical Phase III, randomized, global, multicenter, double-blind, active-controlled trials evaluating ublituximab compared to teriflunomide in patients with RMS Efficacy.


On April 15 this year, based on updated overall survival (OS) data from the Phase III UNITY-CLL trial.

TG has voluntarily withdrawn the Biologics License Application (BLA)/Supplemental New Drug Application (sNDA) for the U2 combination Ukoniq (umbralisib) in combination with ublituximab in adult patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).

And voluntarily stopped selling Ukoniq, which meant the company’s only product was withdrawn from the market. TG later said it would focus its resources on multiple sclerosis and autoimmunity platforms in the future.

The original PDUFA date for Ublituximab was September 28, 2022. The extension also sent TG shares down 14.51%.




Drug name: Valoctocogene Roxaparvovec

Company Name: BioMarin

Indications: Hemophilia A

On May 31, BioMarin announced that it had recently received a response letter from the FDA requesting that it provide additional data when it plans to submit a BLA for valoctocogene roxaparvovec, a hemophilia A therapy.


This AAV5 vector-based gene therapy is currently under review by the European Medicines Regulatory Agency for the treatment of severe hemophilia A. Last month, BioMarin announced plans to resubmit the BLA to the FDA in June 2022.


The company said that based on recent feedback from the FDA on the BLA program, the FDA requested that additional information and data analysis be included in the BLA prior to submission. The FDA did not require additional preclinical or clinical studies. BioMarin expects to resubmit the BLA by the end of September.


In August 2020, the FDA rejected approval of valoctocogene roxaparvovec for the treatment of hemophilia A.




Drug Name: ONS-5010/Lytenava

Company Name: Outlook Therapeutics

Indications: Wet age-related macular degeneration

On May 31, Outlook, Inc. announced that the FDA is requesting additional information to complete the BLA submission for ONS-5010/Lytenava (bedalizumab biosimilar) for the treatment of wet age-related macular degeneration (wet AMD) .

Outlook said it has voluntarily withdrawn the BLA for ONS-5010 and is actively responding to the FDA’s request. The company plans to resubmit the revised BLA by September 2022.


ONS-5010 is an investigational bevacizumab ophthalmic formulation administered by intravitreal injection for the treatment of wet AMD and other retinal diseases.

Because there are currently no approved ophthalmic formulations of bevacizumab, clinicians using bevacizumab in the clinical setting of retinal patients must use unapproved repackaged bevacizumab injections supplied by the dispensing pharmacy,

These products carry a risk of contamination and their potency and efficacy may be inconsistent. If approved, ONS-5010 could replace unapproved repackaged bevacizumab from pharmacies, thus fulfilling a need for the treatment of wet AMD.


Outlook submitted a BLA to the FDA in March 2022, based on an aggregation of data from its previously completed clinical trials (NORSE ONE, NORSE TWO, and NORSE THREE).

NORSE ONE is a proof-of-concept and clinical experience trial that helped validate the NORSE TWO (Pivotal Safety and Efficacy Trial) protocol and approach.

The NORSE TWO data were statistically significant and clinically relevant for the primary endpoint and all secondary endpoints.

NORSE THREE is an open-label supplemental safety trial designed to ensure that a sufficient number of patients are dosed with ONS-5010 (bevacizumab for ophthalmic use) to support registration submissions.




3 new drug applications have been delayed by the FDA: Ublituximab, ONS-5010…

(source:internet, reference only)

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