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Rare Diseases

Rare Diseases is monitored by UniPharma because shifts in demand, reimbursement, regulation, competition, or supply priorities can reshape procurement and launch planning.

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Latest articles on Rare Diseases

April 29, 2026

Italian Drugmaker Chiesi to Acquire KalVista for $1.9 Billion

Chiesi's $1.9B acquisition of KalVista signals a strategic expansion in rare immunology, with implications for rare disease treatment supply and commercialization.

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April 29, 2026

Chiesi Group Grows Again in Rare Disease With $1.9B KalVista Acquisition

Chiesi acquires KalVista for $1.9B, adding oral HAE drug Ekterly. The deal boosts rare disease portfolio with a differentiated on-demand treatment.

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April 29, 2026

Chiesi's $1.9bn KalVista Acquisition Bolsters Rare Disease Portfolio with First Oral HAE Therapy

Chiesi acquires KalVista for $1.9bn, securing first oral HAE therapy Ekterly. The deal transforms HAE attack treatment, shifting from injectables to oral, with implications for supply chain, hospital formularies, and market access in UK and EU.

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April 29, 2026

Chiesi's $1.9B Acquisition of KalVista Boosts Rare Disease Portfolio with EKTERLY (sebetralstat) for HAE

Chiesi Group acquires KalVista for $1.9B, gaining oral HAE drug EKTERLY, expanding US presence and targeting €6B revenue by 2030.

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April 29, 2026

Chiesi’s $1.9B KalVista Buyout Bolsters Rare Disease Pipeline

Chiesi's $1.9 billion acquisition of KalVista strengthens its rare disease pipeline and signals consolidation in the orphan drug space.

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April 29, 2026

Chiesi Acquires KalVista for $1.9bn, Adding Oral HAE Drug Sebetralstat

Chiesi’s $1.9bn acquisition of KalVista brings sebetralstat, the first oral on-demand therapy for hereditary angioedema, expanding its rare disease pipeline.

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April 29, 2026

Chiesi Acquires KalVista for $1.9B, Adding Oral HAE Therapy Ekterly

Chiesi acquires KalVista for $1.9B, gaining oral HAE therapy Ekterly. The deal reshapes the rare disease competitive landscape and expands Chiesi's U.S. presence.

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April 29, 2026

Chiesi Group Acquires KalVista for $1.9 Billion to Boost Rare Disease Portfolio

Chiesi's $1.9 billion acquisition of KalVista strengthens its rare disease pipeline, signaling continued consolidation in the space.

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April 29, 2026

Chiesi Acquires KalVista for $1.9B to Strengthen Rare Disease Portfolio

Chiesi Group buys KalVista for $1.9B, adding oral HAE drug EKTERLY (sebetralstat) to rare disease portfolio. Deal expected to close Q3 2026.

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April 29, 2026

Chiesi's $1.9 Billion KalVista Deal Puts Rare Diseases First

Chiesi's $1.9B cash buyout of KalVista adds oral HAE drug EKTERLY and boosts US rare-disease footprint as it targets €6B revenue by 2030.

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April 29, 2026

Chiesi Group Acquires KalVista Pharmaceuticals, Expanding Rare Disease Portfolio with Oral HAE Therapy

Chiesi's $1.9bn acquisition of KalVista brings the first oral on-demand HAE therapy, sebetralstat, strengthening its rare disease portfolio and US commercial infrastructure.

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April 29, 2026

From constraint to catalyst: innovation in rare disease clinical development

Rare disease development forces innovation in trial design and evidence generation. FDA flexibility on single pivotal trials reflects a shift with implications for manufacturers entering this space.

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April 29, 2026

AI is finally cracking rare disease diagnosis and that could save years of searching

EvORanker identifies the correct disease-causing gene as top candidate in nearly 70% of cases, potentially cutting years off rare disease diagnosis.

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April 28, 2026

Rocket sells priority review voucher for $180M after Kresladi gene therapy approval

Rocket Pharmaceuticals sold a rare pediatric disease priority review voucher for $180 million after FDA approval of its gene therapy Kresladi.

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April 27, 2026

Intellia's CRISPR Therapy Shows Efficacy in HAE Phase 3 Trial

A single dose of lonvo-z reduced swelling attacks in HAE patients, positioning it as the second approved CRISPR medicine and first in vivo treatment. What this means for market access.

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April 27, 2026

Intellia's CRISPR Treatment Reduces Swelling Attacks in Hereditary Angioedema Trial

Intellia's CRISPR-based therapy for hereditary angioedema cut attacks by 87% in a Phase 3 trial, paving the way for FDA approval as the first in vivo gene editing medicine.

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April 27, 2026

Breaking Barriers in Rare Disease Clinical Trials: Lessons from the VALOR Trial

Priovant's VALOR trial in dermatomyositis offers lessons on study design, patient retention, and sponsor-vendor collaboration for rare disease phase 3 trials.

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