Please login to the form below

Not currently logged in

Novartis receives FDA breakthrough therapy designation for rare disease med

The Swiss pharma company is eyeing approval in PNH and C3G indications

Swiss pharma company Novartis has received a breakthrough therapy designation (BTD) for its experimental medication iptacopan for the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).

The therapy, also known as LNP023, was also awarded a rare paediatric disease designation at the same time for the treatment of C3 glomerulopathy (C3G), a group of ultra rare conditions that can cause end-stage renal disease.

Iptacopan is an oral factor B inhibitor and is in development not only for PNH and C3G, but also a number of other renal conditions with complement system involvement.

This includes IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN).

The BTD designation was based on the positive interim results from two ongoing phase 2 studies, within which iptacopan demonstrated benefits in patients who remained anaemic and dependent on transfusions, despite standard of care anti-complement treatment, as well as monotherapy in anti-C5 naive PNH patients.

In this study, iptacopan significantly reduced proteinuria – a marker of inflammation in the kidneys – by 49% compared to baseline values at 12 weeks, in 12 patients with C3G.

In addition, iptacopan stabilised renal function as assessed by eGFR (estimated glomerular filtration rate) at week 12 and was maintained in the seven patients who were treated for a total of six months.

“Iptacopan is the most advanced asset in our nephrology pipeline.” said John Tsai, head of global drug development and chief medical officer at Novartis.

“This data demonstrates that it has the potential to improve the lives of patients with C3G,” he added.

If approved under the accelerated pathway, Novartis is likely to compete with Alexion’s C5-targeting therapy Soliris (eculizumab), which became the first treatment for all patients with PNH and atypical haemolytic uremic syndrome (aHUS) in 2007.

Alexion’s other long-acting C5 inhibitor, Ultomiris (ravulizumab‑cwvz) is also approved in these indications, making the franchise Novartis potential main competitors.

A BTD helps to expedite the development and review of medicines for serious conditions with unmet medical need, where a therapy has promising early-stage data to support the review.

The European Medicines Agency (EMA) has also granted iptacopan a PRIME designation in C3G, as well as an orphan drug designation for the therapy in IgA nephropathy (IgAN).

According to Novartis, iptacopan 'has the potential to become the first alternative complement pathway inhibitor to slow disease progression in a number of complement driven diseases'.

Article by
Lucy Parsons

17th December 2020

From: Regulatory



PMEA Awards 2020

COVID-19 Updates and Daily News

Featured jobs


Add my company
3 Monkeys Zeno

3 Monkeys Zeno is an award winning global creative communications consultancy – home to a collective of creative and strategic...

Latest intelligence

PME Jan cover
AstraZeneca’s focus on diagnosing and managing chronic kidney disease
People living with chronic kidney disease are in urgent need of new and improved approaches to diagnosis and care...
Delphi Technique Virtual Co-creation: A Customer Story
How a client leveraged the Delphi technique to co-create a consensus statement and algorithm using virtual touchpoints....
AI healthcare
Three ways AI will change healthcare in the next decade
Looking forward, there is no doubt that AI will continue to take centre stage in global healthcare initiatives...