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
US biotech ReAlta Life Sciences has appointed Howard Berman as chairman and chief executive officer and Kia Motesharei as president and chief operating officer, positioning the company for a more intensive development phase around its lead asset pegtarazimod. 24 March 2026
Biotechnology
Swiss pharma giant Roche says that it has made the difficult decision not to advance emugrobart—an investigational anti-myostatin antibody, also known as GYM329—into Phase III development in facioscapulohumeral muscular dystrophy (FSHD). 23 March 2026
Pharmaceutical
US biotech Rhythm Pharmaceuticals has secured US regulator approval for Imcivree (setmelanotide) in acquired hypothalamic obesity, marking the first approved treatment for the rare condition characterized by rapid weight gain and severe hunger. 23 March 2026
Pharmaceutical
UK pharma major GSK has announced that the US Food and Drug Administration (FDA) has approved Lynavoy (linerixibat) for the treatment of cholestatic pruritus in adult patients with PBC. 20 March 2026
Pharmaceutical
US rare disease drug developer Sarepta Therapeutics
today provided an update on its ongoing regulatory interactions with the US Food and Drug Administration (FDA) regarding Amondys 45 (casimersen) and Vyondys 53 (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD). 19 March 2026
Pharmaceutical
Latin America has never been short of markets that look promising on paper and frustrating in practice. Brazil still belongs in both camps. It is the region’s largest pharmaceutical market, one of the state’s biggest spending arenas, and a country where the regulator now carries more weight than many multinationals once assumed. 19 March 2026
Biotechnology
Swedish Orphan Biovitrum, the Nordic biotech company also known as Sobi, today announced that top-line results from the Phase IIa EMBRACE study, evaluating Gamifant (emapalumab) in patients with interferon-gamma (IFNγ)-driven sepsis (IDS), were presented at the International Symposium on Intensive Care and Emergency Medicine (ISICEM) by Professor Evangelos Giamarellos-Bourboulis, president of the Hellenic Institute for the Study of Sepsis. 18 March 2026
Pharmaceutical
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Sanofi’s venglustat, a novel, investigational oral glucosylceramide synthase inhibitor, to treat neurological manifestations of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder. 18 March 2026
Pharmaceutical
Sentynl Therapeutics, a USA-based biopharma and wholly-owned subsidiary of India’s Zydus Lifesciences, has announced a deal with Korean rare genetic diseases specialist PRG S&T to license its investigational molecule progerinin (SLC-D011) for Hutchinson-Gilford progeria syndrome (HGPS). 16 March 2026
Pharmaceutical
armaceutical Development & Commercialization and its parent Japan-based Otsuka Pharmaceutical have announced new open-label extension study data for repinatrabit (JNT-517), demonstrating early and clinically-meaningful reductions in blood phenylalanine (Phe) levels (-67% mean change from baseline) in adolescents with phenylketonuria (PKU).
13, 2026 13 March 2026
Biotechnology
US biotech Regenxbio has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, its gene therapy for Duchenne muscular dystrophy (DMD). 13 March 2026
Biotechnology
Ultragenyx Pharmaceutical announced positive results from its Phase III Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. 13 March 2026
Pharmaceutical
US drugmaker BioMarin Pharmaceutical today announced positive new data from studies of Voxzogo (vosoritide) in children with achondroplasia, also called dwarfism, in ongoing clinical trials and real-world studies. 12 March 2026
Pharmaceutical
US biotech BridgeBio reported positive interim Phase III data for BBP-418 in limb-girdle muscular dystrophy, showing improvements across several functional measures and a safety profile comparable with placebo. 12 March 2026
Pharmaceutical
Swiss specialty drugmaker Santhera Pharmaceuticals today provides a trading update for the fiscal year 2025. The firm’s share dipped 3.2% to 16.50 francs following the announcement. 12 March 2026
Biotechnology
Swiss–American biotech CRISPR Therapeutics today announced the pricing of $550 million in convertible senior notes due in 2031. The notes are being offered in a private offering to qualified institutional buyers. 11 March 2026
Biotechnology
US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel antisense oligonucleotide (ASO) given once a year for spinal muscular atrophy (SMA), at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. 11 March 2026
Pharmaceutical
The US Food and Drug Administration (FDA) has approved expanded use of Wellcovorin (leucovorin calcium) tablets to treat cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (-FOLR1). 10 March 2026
Biotechnology
Australia-headquartered CSL Limited broke ground Monday for the $1.5 billion expansion of its manufacturing facility in Kankakee, Illinois, USA. 9 March 2026
Biotechnology
Italy’s Chiesi Group and Israel’s Protalix BioTherapeutics have announced that the European Commission (EC) has approved the 2mg/kg every-4-weeks (E4W) dosing regimen for Elfabrio (pegunigalsidase alfa) in adults living with Fabry disease who are stable with an ERT. 9 March 2026
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