Rare Diseases

Sanofi Revels In More Positive Rilzabrutinib Data

 
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The oral BTK inhibitor impresses in a Phase II trial for IgG4-related disease.

Biogen Lays Out Cadence For Felzartamab Data In Kidney Diseases

 

The first Phase III data for the CD38-targeting antibody is expected in 2027 in antibody-mediated rejection in kidney transplant patients, followed by other indications.

Vigil’s Iluzanebart Disappoints In Rare Neurodegenerative Disease

 

The Phase II TREM2 agonist is not part of Sanofi’s planned acquisition.

Sanofi Plays It Safer With Blueprint Buy After Bumpy 2025

 
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But the French major is still keen on early-stage M&A.


Roivant Staying ‘Patient’ On Deals, Leaving Investors Waiting

 

CEO Matt Gline talked to Scrip in an interview about dealmaking and the road back to commercialization.

InflaRx Stock Sinks As Vilobelimab Fails Phase III Skin Trial

 
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A study of the German biotech's intravenous complement factor C5a antibody for pyoderma gangrenosum was stopped for futility.

Sweden’s Hansa Slims Down Ahead of Key Readouts

 
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New CEO Renée Aguiar-Lucander is marking her mark.

Zydus To Continue Mirabegron Sales, Looks To World-First Dual Shigella-Typhoid Vaccine

 
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Zydus expects strong mirabegron sales in FY26 amid US litigation even as it builds a growth pillar in vaccines with a world-first, Gates Foundation-aided dual shigella-typhoid vaccine under development and others on the WHO prequalified list


Lundbeck Lifted By Leap In Migraine Therapy Sales

 
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Vyepti revenues rise by nearly 70% in the first quarter.

Azafaros Banks Over €130m To Advance Nizubaglustat Into Phase III

 
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The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.

J&J’s Imaavy Approval Is The Starting Line For Pipeline-In-A-Product Strategy

 

The company’s FcRn inhibitor nipocalimab was approved by the US FDA for generalized myasthenia gravis (gMG), where it will join a competitive market.

In Brief: Ferrer Signs Deal For Prilenia’s Pridopidine In Europe

 

The companies will co-develop the drug for additional indications beyond Huntington’s disease, while Prilenia retains rights in key global markets.


Merck KGaA Seals SpringWorks Buy

 
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The German group is paying $3.9bn to get hold of the US firm and its two approved products.

Spruce Pivots After Pipeline Failure With BioMarin Tralesinidase Deal

 

After dropping its lead program in December, Spruce has been exploring strategic options – and thinks it has a de-risked path for MPS IIIB therapy tralesinidase from BioMarin.

Glycomine Funds Phase IIb Rare Disease Trial With $115m Series C

 
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Lead drug GLM101 will move into its first placebo-controlled trial based on positive results in adults and adolescents with PMM2-CDG in an open-label Phase IIa study.

New Data Strengthen Case For RemeGen’s Telitacicept In gMG

 

Results from a China Phase III trial show the potential of RemeGen's fusion protein telitacicept in generalized myasthenia gravis. But study design differences make direct comparisons with argenx's FcRn inhibitor Vyvgart challenging.


Vocal For ‘Glocal’: How Menarini Is Adapting To Outperform In The Asia Pacific

 

Menarini's Asia-Pacific CEO talks to Scrip about how the Italian group is tailoring its portfolio and pricing approach to local markets making strong strides in primary care and consumer health, while also charting a distinct path in rare diseases and oncology. The China market is also a key focus including as a potential source of innovation as is partnering, where the group has a rich history.

Orphans Cling On To Growth Advantage As Market Share Heads Towards 20%

 

It might be the beginning of the end for the orphan drugs party but there is still sales growth enjoyment to be had for the sector, whose star performers are now looking increasingly like mainstream drugs.

Sanofi Set To Shake Up Crowded Hemophilia Space After Qfitlia Approval

 
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The small interference RNA therapeutic can be used by patients regardless of inhibitor status.

RemeGen Fast-Tracks Potential World-First BLys/APRIL Agent For gMG In Global Phase III Trial

 

RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.