Rare Diseases
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Pharmaceutical
New data published in Brain Communications offers a strong picture of how devastating thymidine kinase 2 deficiency (TK2d) can be without treatment - and how vital earlier diagnosis and treatment are in potentially changing the course of disease. 17 June 2026
Biotechnology
Polish biotech Mabion has signed a letter of intent with French TechDev company Oddifact to explore MabionCD20 in rare diseases, reopening a program that was once developed as a rituximab biosimilar. 17 June 2026
Biotechnology
San Diego, USA-based Soleno Therapeutics, which has just been acquired by Neurocrine Biosciences, has announced late-breaking data at ENDO 2026 showing that resuming treatment with Vykat XR (diazoxide choline) extended-release tablets . 16 June 2026
Biotechnology
Shares of Australian biotech Dimerix leapt 21.2% to A$0.20 today on the news of a landmark licensing deal. 16 June 2026
Biotechnology
China-based Fosun Pharmaceutical announced that its self-developed MEK 1/2 selective inhibitor luvometinib tablets (trade name: Fumaining) has been officially approved by China’s National Medical Products Administration (NMPA). 16 June 2026
Pharmaceutical
US biopharma Neurocrine Biosciences has reported new two-year data for Crenessity (crinecerfont), showing sustained reductions in disease biomarkers, lower glucocorticoid dosing and improvements in clinical measures among children and adolescents with classic congenital adrenal hyperplasia (CAH). 15 June 2026
Biotechnology
US CRISPR gene editing company Intellia Therapeutics over the weekend presented additional positive results from the global Phase III HAELO clinical trial of lonvoguran ziclumeran (lonvo-z; formerly NTLA-2002) for hereditary angioedema (HAE). 15 June 2026
Biotechnology
M&A featured heavily last week, with Incyte announcing plans to buy Vega Therapeutics, a subsidiary of Star Therapeutics, for up to $2 billion. Then came UK pharma major GSK’s revelation of an agreement to acquire US firm Nuvalent for up to $10.6 billion. Also of note, US biotech Cartesian Therapeutics entered into a licensing deal with China’s WestGene Biopharma on CAR-T therapies for autoimmune diseases. Sweden’s Camurus was served another complete response letter (CRL) by the US Food and Drug Administration (FDA) on its filing for Oclaiz (CAM2029). 14 June 2026
Pharmaceutical
Shares in Sweden based drug developer Camurus fell as much as 7% on Thursday, after the US Food and Drug Administration (FDA) rejected the company’s application for Oclaiz (octreotide; CAM2029) extended-release injection a treatment for acromegaly. 12 June 2026
Biotechnology
Swiss pharma Novartis said its investigational RNA therapy del-brax has met the primary and key secondary endpoints in a Phase I/II study of facioscapulohumeral muscular dystrophy, supporting the dose already being tested in a Phase III trial. 12 June 2026
Pharmaceutical
An interview with Anna Petryk, Executive Global Medical Director who leads the global medical affairs team in the Rare Bone and Endocrine Disorders therapeutic area at Alexion, AstraZeneca Rare Disease. 11 June 2026
Biotechnology
US biotech Beren Therapeutics has lined up a financing package worth up to $300 million to support preparations for the potential launch of adrabetadex, its investigational treatment for infantile-onset Niemann-Pick disease type C. 11 June 2026
Biotechnology
San Francisco-based biotech SonoThera today announced the closing of an oversubscribed $125 million Series B financing round to advance its lead programs in Duchenne muscular dystrophy (DMD) and autosomal dominant polycystic kidney disease (ADPKD) into the clinic and expand its pipeline of targeted, redosable genetic medicines. 10 June 2026
Pharmaceutical
French drugmaker Servier has entered a strategic collaboration with the USA-based n-Lorem Foundation to advance research into rare neurodevelopmental disorders using antisense oligonucleotide (ASO) technology. 10 June 2026
Biotechnology
Swedish rare diseases company Hansa Biopharma today announced that Fredrik Röök has been appointed as vice president business development effective August 17. 10 June 2026
Pharmaceutical
The European Federation of Pharmaceutical Industries and Associations (EFPIA) has called for stronger action on rare diseases, warning that fragmented systems continue to hold back research, diagnosis and access to treatment. 8 June 2026
Pharmaceutical
Among licensing news of note last week, US drug developer Agios Pharma struck a deal with South Korea’s Oscotec for the latter’s cevidoplenib, a potential treatment for immune thrombocytopenia. US major AbbVie won European Commission approval for its migraine drug Aquipta. Travere Therapeutics licensed rights to civorebrutinib from China’s Everest Medicines, and Alnylam Pharmaceuticals entered into an AI collaboration with Inceptive Nucleics worth a potential $2 billion. 7 June 2026
Pharmaceutical
Chiesi Global Rare Diseases, a unit of privately-held Italian drugmaker Chiesi, announced that the European Commission (EC) has approved Lojuxta (lomitapide) capsules for use in children 5 years of age and older with homozygous familial hypercholesterolemia (HoFH), for use alongside diet and other lipid-lowering treatments, including LDL-apheresis where available. 6 June 2026
Generics
Russia will have its own generic of one of the world’s most expensive drug against cystic fibrosis, as the Russian pharmaceutical company Amedart announced its plans launch a domestic analogue of Trikafta (elexacaftor/tezacaftor/ivacaftor), a drug from the American company Vertex Pharmaceuticals for the treatment of cystic fibrosis, reports The Pharma Letter’s local correspondent. 5 June 2026
Biotechnology
US biotech Biogen has received US Food and Drug Administration (FDA) Breakthrough Therapy designation for salanersen, its investigational antisense oligonucleotide for spinal muscular atrophy (SMA). 5 June 2026
Pharmaceutical
A feature on the amyloidosis space. 4 June 2026
Pharmaceutical
Colorado, USA-based Edgewise Therapeutics, a muscle disease biopharmaceutical company, announced that it has entered into a definitive agreement with French independent pharma company Servier. 2 June 2026
Pharmaceutical
Cambridge, USA-based drug developer Agios Pharmaceuticals has announced an agreement with Oscotec, a clinical-stage drug discovery and development company focused on immunology and oncology, headquartered in South Korea, to license the exclusive global rights to cevidoplenib, a novel, next-generation, oral spleen tyrosine kinase (SYK) inhibitor. 2 June 2026
Biotechnology
USA-based rare disease biotech Rallybio has agreed to acquire privately-held Avenzo Therapeutics in a reverse merger that will shift the public company’s focus to oncology. 2 June 2026
Biotechnology
Cambridge, USA-based Oak Hill Bio, a clinical-stage rare disease therapeutics company, today announced the closing of a $32.5 million Series A financing. 1 June 2026
Pharmaceutical
US pharma major Eli Lilly’s Verzenio (abemaciclib) significantly delayed disease progression in patients with advanced dedifferentiated liposarcoma, according to Phase III SARC041 data presented at the American Society of Clinical Oncology (ASCO) Annual Meeting. 1 June 2026
Biotechnology
The hereditary angioedema (HAE) pharmaceutical landscape is evolving at an unprecedented speed. Following a surge of approvals in recent years, the market is now facing a late-stage development bottleneck, as 46% of pipeline drugs are currently in Phase III. 1 June 2026
Biotechnology
US biotech Capricor Therapeutics just named Michael Maurer as Chief Commercial Officer as the company heads toward an August 22 PDUFA date for deramiocel, its cell therapy for Duchenne muscular dystrophy. 30 May 2026
Biotechnology
UK pharma major AstraZeneca today disclosed that the global CARES Phase III clinical program showed that treatment with anselamimab, a potential first-in-class anti-fibril therapy, resulted in nominally statistically significant and highly clinically meaningful benefit in adults with advanced kappa light chain (AL) amyloidosis as first-line therapy added to standard of care plasma cell dyscrasia (PCD) treatments, compared to placebo. 29 May 2026
Pharmaceutical
The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for Sanofi’s venglustat, a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), for the treatment of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder. 28 May 2026
Pharmaceutical
French drugmaker Ipsen today announced the presentation of new late-breaking results from the ELATIVE Phase III trial and two real-world studies at the European Association for the Study of the Liver (EASL) congress. 28 May 2026
Biotechnology
US biotech Secretome Therapeutics has raised $30 million in series A financing from RA Capital Management, giving the Texan company fresh backing for a neonatal cardiac cell therapy pipeline led by STM-01. 28 May 2026
Biotechnology
The US Food and Drug Administration has approved US pharma AbbVie’s Decnupaz (pivekimab sunirine-pvzy) for adults with blastic plasmacytoid dendritic cell neoplasm, a rare and aggressive blood cancer. 28 May 2026
Biotechnology
US biotech Immunic today announced the appointment of distinguished biopharmaceutical executive Erik Lundgren as chief executive (CEO), effective May 22, 2026, with employment beginning on June 1, 2026. 27 May 2026
Biotechnology
USA-based Immix Biopharma, a global leader in AL amyloidosis, has announced the closing of its previously announced underwritten registered offering of 16,778,524 shares of its common stock at a public price of $8.94 per share. 26 May 2026
Pharmaceutical
Important research news last week included Regeneron releasing disappointing Phase III data for its investigational drug fianlimab as a treatment for metastatic melanoma and positive results for Relay Therapeutics’ zovegalisib in vascular anomalies. Additionally, BioMarin released Phase III data on its hypochondroplasia drug Voxzogo. Also of note, SERB Pharmaceuticals acquired rights to Swedish firm Hansa Biopharma’s Idefirix with an upfront payment of $128 million. 24 May 2026
Pharmaceutical
US biotech Amgen and Japanese partner Kissei Pharmaceutical have restored the use of Tavneos (avacopan) in new patients in Japan after regulators ordered stronger warnings around serious liver injury, including fatal cases. 22 May 2026
Pharmaceutical
US rare disease drugmaker BioMarin said its growth disorder therapy Voxzogo (vosoritide) met the primary endpoint in a pivotal Phase III study in children with hypochondroplasia, potentially opening the way for the first approved treatment for the condition. 21 May 2026
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has launched a public consultation on its proposed Rare Disease Therapies Framework designed to accelerate development and approval of treatments for rare and ultra-rare conditions. 21 May 2026
Pharmaceutical
Family-controlled Italian drugmaker Chiesi Group has announced a planned leadership transition, appointing Maria Paola Chiesi as chair of its board of directors, succeeding Alessandro Chiesi, who will become vice chair. 20 May 2026
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