Rare Diseases
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Pharmaceutical
The local unit of US biotech major Biogen today announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorization to the high dose regimen (50/28mg) of Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). 14 April 2026
Biotechnology
US biotech major Regeneron Pharmaceuticals and Australian radiopharmaceuticals specialist Telix Pharmaceuticals have announced a collaboration, sending the latter company’s shares up by 8% in Monday’s trading. 13 April 2026
Generics
The Russian Ministry of Health has begun more active cancelling of tenders for drug procurements, waiting for the launch of cheaper generics, as the federal budget experiences a shortage of funds for procurements of expensive original drugs. 13 April 2026
Pharmaceutical
The UK’S Medicines and Healthcare products Regulatory Agency (MHRA) has approved Swedish Orphan Biovitrum Tryngolza (olezarsen) to help treat adults with familial chylomicronemia syndrome (FCS). 11 April 2026
Pharmaceutical
US specialty pharma company Cosette Pharmaceuticals announced the closing of a definitive agreement to acquire the US sales and distribution rights for a portfolio of seven branded products from Assertio Holdings. 9 April 2026
Biotechnology
Late last year, Julie Dehaene-Puype stepped into the role of Regional President at Kyowa Kirin International (KKI), a specialty pharma developing innovative treatments for rare and genetic diseases, including bone and mineral disorders, rare blood cancers and other inherited genetic conditions. 8 April 2026
Biotechnology
Rare disease drug pricing has long rested on a simple trade-off. Small patient populations and high development costs justified higher prices, and payers largely accepted that logic because the overall budget impact remained limited. That balance is becoming harder to maintain as orphan drugs now account for a growing share of new launches and an increasing portion of drug spending. 8 April 2026
Biotechnology
German drug developer Evotec reported fourth-quarter 2025 revenue of $293 million, up 14%, and adjusted EBITDA of $67 million, up 103%, driven largely by a license payment linked to the sale of its Toulouse biologics site to Sandoz. 8 April 2026
Biotechnology
Syneron Bio, a China based leader in intelligent platform-driven macrocyclic peptide drug discovery, announced the successful closing of its Series B financing on March 31. The news comes less than four months after Syneron closed its Series A and A+ rounds at close to $100 million in total. 7 April 2026
Generics
India has moved ahead in developing indigenous capacity for cell and gene therapies, with regulatory bodies issuing national guidelines to ensure safe and ethical deployment of CRISPR-based technologies. 3 April 2026
Pharmaceutical
US drugmaker Vertex Pharmaceuticals today announced the US Food and Drug Administration (FDA) has approved expanded indications for two of its top-selling drugs. 1 April 2026
Biotechnology
The US Food and Drug Administration (FDA) is alerting patients and health care professionals about serious post-marketing cases, including fatal cases, of drug-induced liver injury (DILI) associated with US biotech major Amgen’s Tavneos (avacopan). 1 April 2026
Pharmaceutical
The efzimfotase alfa (ALXN1850) Phase III clinical program, designed to study a broad hypophosphatasia (HPP) patient population, demonstrated positive results, UK pharma major AstraZeneca announced today. 31 March 2026
Pharmaceutical
The European Commission has granted a conditional marketing authorization for French pharma major Sanofi’s Rezurock (belumosudil) for the treatment of chronic graft-versus-host disease (GVHD) in adults and in children aged 12 years and older with a body weight of at least 40 kg. 31 March 2026
Pharmaceutical
Belgian biopharma UCB has secured European Commission approval for Kygevvi (doxecitine, doxribtimine), the first authorized treatment for thymidine kinase 2 deficiency, a rare and often fatal mitochondrial disease. 31 March 2026
Biotechnology
March 2026 marked a significant inflection point for rare disease therapeutics, as regulatory momentum, policy pressure, and scientific innovation converged. 30 March 2026
Pharmaceutical
US pharma major Merck is moving Winrevair (sotatercept-csrk) toward a Phase III study in combined post- and precapillary pulmonary hypertension linked to heart failure with preserved ejection fraction, betting the drug can open a second growth market beyond its first pulmonary arterial hypertension use. 30 March 2026
Biotechnology
US biotech Biogen has announced positive results from the Phase II part of the AMETHYST Phase II/III study (Part A) of litifilimab in people living with cutaneous lupus erythematosus (CLE). 30 March 2026
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I), US biotech Rocket Pharmaceuticals announced today. 27 March 2026
Pharmaceutical
Italian drugmaker Recordati has confirmed it received a non-binding takeover approach from private equity firm CVC Capital Partners, valuing the company at 10.9 billion euros ($12.6 billion). 27 March 2026
Pharmaceutical
The local unit of US biotech major Biogen today announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorization to the high dose regimen (50/28mg) of Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). 14 April 2026
Biotechnology
US biotech major Regeneron Pharmaceuticals and Australian radiopharmaceuticals specialist Telix Pharmaceuticals have announced a collaboration, sending the latter company’s shares up by 8% in Monday’s trading. 13 April 2026
Generics
The Russian Ministry of Health has begun more active cancelling of tenders for drug procurements, waiting for the launch of cheaper generics, as the federal budget experiences a shortage of funds for procurements of expensive original drugs. 13 April 2026
Pharmaceutical
The UK’S Medicines and Healthcare products Regulatory Agency (MHRA) has approved Swedish Orphan Biovitrum Tryngolza (olezarsen) to help treat adults with familial chylomicronemia syndrome (FCS). 11 April 2026
Pharmaceutical
US specialty pharma company Cosette Pharmaceuticals announced the closing of a definitive agreement to acquire the US sales and distribution rights for a portfolio of seven branded products from Assertio Holdings. 9 April 2026
Biotechnology
Late last year, Julie Dehaene-Puype stepped into the role of Regional President at Kyowa Kirin International (KKI), a specialty pharma developing innovative treatments for rare and genetic diseases, including bone and mineral disorders, rare blood cancers and other inherited genetic conditions. 8 April 2026
Biotechnology
Rare disease drug pricing has long rested on a simple trade-off. Small patient populations and high development costs justified higher prices, and payers largely accepted that logic because the overall budget impact remained limited. That balance is becoming harder to maintain as orphan drugs now account for a growing share of new launches and an increasing portion of drug spending. 8 April 2026
Biotechnology
German drug developer Evotec reported fourth-quarter 2025 revenue of $293 million, up 14%, and adjusted EBITDA of $67 million, up 103%, driven largely by a license payment linked to the sale of its Toulouse biologics site to Sandoz. 8 April 2026
Biotechnology
Syneron Bio, a China based leader in intelligent platform-driven macrocyclic peptide drug discovery, announced the successful closing of its Series B financing on March 31. The news comes less than four months after Syneron closed its Series A and A+ rounds at close to $100 million in total. 7 April 2026
Generics
India has moved ahead in developing indigenous capacity for cell and gene therapies, with regulatory bodies issuing national guidelines to ensure safe and ethical deployment of CRISPR-based technologies. 3 April 2026
Pharmaceutical
US drugmaker Vertex Pharmaceuticals today announced the US Food and Drug Administration (FDA) has approved expanded indications for two of its top-selling drugs. 1 April 2026
Biotechnology
The US Food and Drug Administration (FDA) is alerting patients and health care professionals about serious post-marketing cases, including fatal cases, of drug-induced liver injury (DILI) associated with US biotech major Amgen’s Tavneos (avacopan). 1 April 2026
Pharmaceutical
The efzimfotase alfa (ALXN1850) Phase III clinical program, designed to study a broad hypophosphatasia (HPP) patient population, demonstrated positive results, UK pharma major AstraZeneca announced today. 31 March 2026
Pharmaceutical
The European Commission has granted a conditional marketing authorization for French pharma major Sanofi’s Rezurock (belumosudil) for the treatment of chronic graft-versus-host disease (GVHD) in adults and in children aged 12 years and older with a body weight of at least 40 kg. 31 March 2026
Pharmaceutical
Belgian biopharma UCB has secured European Commission approval for Kygevvi (doxecitine, doxribtimine), the first authorized treatment for thymidine kinase 2 deficiency, a rare and often fatal mitochondrial disease. 31 March 2026
Biotechnology
March 2026 marked a significant inflection point for rare disease therapeutics, as regulatory momentum, policy pressure, and scientific innovation converged. 30 March 2026
Pharmaceutical
US pharma major Merck is moving Winrevair (sotatercept-csrk) toward a Phase III study in combined post- and precapillary pulmonary hypertension linked to heart failure with preserved ejection fraction, betting the drug can open a second growth market beyond its first pulmonary arterial hypertension use. 30 March 2026
Biotechnology
US biotech Biogen has announced positive results from the Phase II part of the AMETHYST Phase II/III study (Part A) of litifilimab in people living with cutaneous lupus erythematosus (CLE). 30 March 2026
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I), US biotech Rocket Pharmaceuticals announced today. 27 March 2026
Pharmaceutical
Italian drugmaker Recordati has confirmed it received a non-binding takeover approach from private equity firm CVC Capital Partners, valuing the company at 10.9 billion euros ($12.6 billion). 27 March 2026
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