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GSK’s Benlysta given orphan drug status by FDA for systemic sclerosis

The rare autoimmune disease affects two to ten people per million and has limited treatments


GSK’s Benlysta (belimumab) has been given Orphan Drug Designation (ODD) by the US Food and Drug Administration for the potential treatment of systemic sclerosis (SSc), the company announced.

The antibody will now benefit from the incentives given to those drugs granted orphan status through the FDA’s ODD programme, introduced by the organisation to improve the availability of treatments for rare diseases.

Affecting two to ten people per million, SSc is a rare autoimmune disease that causes unusual growth of connective tissues and can affect a patient’s musculoskeletal system, heart, lungs, kidneys, skin and other organs.

Interstitial lung disease (ILD) is a common and serious complication of SSc, marked by inflammation and scar tissue build-up in the lungs, and is the leading cause of death in patients living with the disease.

The company said in a statement: ‘With limited treatment options available for SSc-ILD, this ODD reflects the need for further research and the potential for belimumab to address a critical need for people living with this debilitating condition.’

It also outlined plans to initiate a phase 2/3 trial of Benlysta for SSc associated with ILD in the first half of this year.

Research indicates that elevated levels of B-lymphocyte stimulator (BLyS) and autoreactive B cells play a central role in the development of SSc.

A human monoclonal antibody, Benlysta inhibits the prolonged survival of B cells induced by increased BLyS, including autoreactive B cells.

It is already approved in the US, the EU and Canada to treat active systemic lupus erythematosus (SLE) – the most common type of lupus – with its initial FDA approval in 2011 making it the first new drug approved to treat lupus in 56 years.

Benlysta also received FDA approval in 2020 for active lupus nephritis – a serious inflammation of the kidneys caused by SLE – after the antibody demonstrated significant improvements in primary efficacy renal response compared to placebo, as well as a decrease in risk for experiencing a renal-related event.

In November 2022, a global survey conducted on behalf of GSK exposed gaps in the treatment of lupus.

It found that of the 648 healthcare professionals surveyed, nearly half believed that patients with the disease had not been receiving 'optimal care' over the past two years due to a range of factors including the COVID-19 pandemic.

Article by
Emily Kimber

1st February 2023

From: Research, Regulatory



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