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rare disease

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Representative image of acromegaly drug development as Camurus’ Oclaiz FDA setback puts rare disease manufacturing risk, long-acting injectable therapies, and U.S. regulatory scrutiny under sharper focus.
Pharma & Biotech

Camurus Oclaiz CRL shows how one manufacturing site can stall a differentiated endocrine therapy

Find out how Camurus’ fresh FDA setback for Oclaiz could delay its U.S. acromegaly launch and reshape rare disease manufacturing risk.

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Representative image: A pediatric neurology research setting highlights the clinical focus on tralesinidase alfa, enzyme replacement therapy, and long-term treatment advances for Sanfilippo syndrome type B.
Pharma & Biotech

Could Spruce Biosciences finally change the Sanfilippo syndrome type B treatment landscape?

Spruce Biosciences’ MPS IIIB data raise a pivotal question: can tralesinidase alfa turn biomarker gains into disease-changing therapy?

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Representative image of a rare disease neurology consultation, illustrating how Sanofi’s venglustat FDA priority review could change treatment options for type 3 Gaucher disease.
Pharma & Biotech

Can Sanofi’s venglustat priority review change the treatment outlook for type 3 Gaucher disease?

Sanofi’s venglustat could target neurological type 3 Gaucher disease. The FDA review now tests whether rare disease care can shift.

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Representative image of a neuromuscular clinic consultation, reflecting Servier’s acquisition of Edgewise Therapeutics’ sevasemten programme and the growing race to develop oral therapies for Becker and Duchenne muscular dystrophy.
Pharma & Biotech

Can Servier’s $2.65bn Edgewise deal reshape the muscular dystrophy drug race?

Servier is buying sevasemten before pivotal muscular dystrophy data. The deal gives Edgewise cash and investors a sharper cardiology story.

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Representative image showing a biotech licensing discussion around long-acting GLP-2 therapy, reflecting Lilly and Hanmi Pharm’s sonefpeglutide deal for short bowel syndrome treatment.
Pharma & Biotech

Can Lilly turn Hanmi’s sonefpeglutide into the next long-acting GLP-2 rare disease bet?

Read why Lilly’s Hanmi sonefpeglutide deal could reshape long-acting GLP-2 therapy for short bowel syndrome.

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Representative image of MRI brain scan analysis as Ono Pharmaceutical’s ONO-2808 trial data highlights new research momentum in multiple system atrophy treatment.
Pharma & Biotech

Why Ono’s Phase 2 MSA data may matter beyond one World Parkinson Congress poster

Ono’s ONO-2808 data hints at disease modification in MSA. See why Phase 3 may decide whether the signal becomes a breakthrough.

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Pharma & Biotech

Neurocrine’s $2.9bn Soleno deal raises the stakes in Prader-Willi syndrome treatment

Find out how Neurocrine’s $2.9bn Soleno acquisition could reshape VYKAT XR’s rare disease launch strategy.

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Representative image of neonatal gene therapy research in a modern clinical setting. iECURE’s ECUR-506 data have sharpened interest in whether targeted gene insertion could reduce hyperammonemic events in infants with neonatal-onset OTC deficiency.
Pharma & Biotech

Is iECURE’s infant gene editing therapy quietly changing the OTC deficiency playbook?

A rare disease gene editing signal is only the start. iECURE now has to prove ECUR-506 can sustain OTC deficiency control.

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Representative image: Angelini Pharma’s $4.1 billion Catalyst Pharmaceuticals acquisition highlights the growing race for rare disease and neurology assets, with Firdapse, Agamree and U.S. commercial scale at the center of the deal.
Pharma & Biotech

Angelini Pharma bets $4.1bn on rare neurology scale with Catalyst Pharmaceuticals

Angelini gains U.S. rare disease scale. Catalyst gets certainty, but Firdapse concentration keeps the real test ahead.

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Representative image: Arrowhead Pharmaceuticals’ REDEMPLO approval in Australia highlights the growing role of RNAi therapies in treating familial chylomicronemia syndrome and severe triglyceride disorders.
Pharma & Biotech

Arrowhead just unlocked Australia for REDEMPLO, but the bigger RNAi test starts now

Arrowhead has regulatory momentum in rare lipid disease. The harder test is turning REDEMPLO approval into durable global uptake.

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