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Castleman’s disease

- PMLiVE

Bristol Myers Squibb’s New Drug Application for mezigdomide accepted by FDA

Mezigdomide improves progression-free survival in patients with relapsed or refractory multiple myeloma

- PMLiVE

Teva submits FDA application for paediatric Tourette syndrome therapy

Ecopipam could become the first new FDA-approved treatment for paediatric Tourette syndrome in over ten years

- PMLiVE

Roche’s Enspryng given FDA Priority Review as thyroid eye disease treatment

This is the first and only at-home subcutaneous treatment option for this disease

- PMLiVE

Regeneron’s New Drug Application for cemdisiran accepted for review by FDA and EMA

Globally, around 150 to 200 out of every million people have myasthenia gravis

- PMLiVE

Roche’s Tecentriq gets FDA Priority Review for specific stage 3 colon cancer

Over one million people are diagnosed globally each year and around 30% of stage 3 patients relapse within five years

- PMLiVE

Lilly’s Ebglyss approved by FDA as maintenance dose for atopic dermatitis

This new option for patients allows for as few as six maintenance injections per year

- PMLiVE

J&J’s Erleada prostate cancer study shows significant reduction in risk of metastasis

Erleada plus hormone therapy significantly improved key short- and long-term clinical outcomes

- PMLiVE

LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

- PMLiVE

FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders

The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns

- PMLiVE

J&J’s Icotyde approved by US FDA for plaque psoriasis

Plaque psoriasis affects over 125 million people worldwide

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

- PMLiVE

PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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