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Pharmaceutical
Italian pharma major Recordati (RECI: MI) has reported strong preliminary consolidated financial results for 2024, with the firm’s shares edging up 2.3% to 60.40 euros. 14 February 2025
Biotechnology
The European Commission (EC) has approved Andembry (garadacimab), the first and only once-monthly treatment targeting factor XIIa to prevent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older. 13 February 2025
Pharmaceutical
The Food and Drug Administration (FDA) has approved a New Drug Application (NDA) from Swiss pharma giant Roche’s Genentech subsidiary for an Evrysdi (risdiplam) tablet for people living with spinal muscular atrophy (SMA). 13 February 2025
Pharmaceutical
Santhera Pharmaceuticals, a Swiss specialty pharma focused on rare diseases, has announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement for Agamreee (vamorolone) to treat Duchenne muscular dystrophy (DMD). 13 February 2025
Biotechnology
Biotech firms Third Harmonic Bio and Inventiva have announced plans to reduce their respective workforces by 50% as part of cost-cutting measures. 12 February 2025
Pharmaceutical
Vertex Pharmaceuticals expects another year of rapid growth in 2025, after achieving this in the fourth quarter and throughout 2024 as a whole. 11 February 2025
Biotechnology
The Roivant subsidiary Kinevant is to discontinue further development of namilumab for the treatment of sarcoidosis. 11 February 2025
Pharmaceutical
Boehringer Ingelheim today revealed that the FIBRONEER-ILD trial of nerandomilast met its primary endpoint, which was the absolute change from baseline in forced vital capacity (FVC) [mL] at week 52 versus placebo. 10 February 2025
Pharmaceutical
The ORPHAN Cures Act (HR 946), a bipartisan bill that removes barriers to treating and curing rare diseases, was reintroduced in the US House of Representative last week. 10 February 2025
Pharmaceutical
Shares of rare disease drug developer X4 Pharmaceuticals took a battering after it said it will restructure its workforce and capital spending. 7 February 2025
Pharmaceutical
Anglo-Swedish pharma major AstraZeneca beat analyst expectations in presenting its impressive latest financial results. 6 February 2025
Biotechnology
California, USA-based rare disease company Ultragenyx Pharmaceutical yesterday released new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically-significant improvement in the Bayley-III raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with Sanfilippo syndrome type A (MPS IIIA) compared to natural history data from untreated patients. 6 February 2025
Biotechnology
Eleva, a German biopharmaceutical company, and 3PBIOVIAN, a global contract development and manufacturing organization (CDMO), have announced a strategic partnership to enhance the production of Eleva's pipeline products. 4 February 2025
Biotechnology
Fondazione Telethon, an Italian biomedical charity focused on rare genetic diseases, has submitted a Marketing Authorization Application (MAA) for the gene therapy etuvetidigene autotemcel to the European Medicines Agency (EMA). 3 February 2025
Pharmaceutical
Boston-based biotech Ironwood Pharmaceuticals (Nasdaq: IRWD) had lost 38% of its market value by Thursday’s close. 31 January 2025
Pharmaceutical
Shares of US drugmaker Vertex Pharmaceuticals jumped 8.4% to $475.00 in after-hours trading yesterday after it announced that the US Food and Drug Administration (FDA) has approved Journavx (suzetrigine), an oral, non-opioid, highly selective NaV1.8 pain signal inhibitor for the treatment of adults with moderate-to-severe acute pain. 31 January 2025
Pharmaceutical
Swiss pharma giant Roche today released full-year 2024 financials, showing that group sales grew 7% at constant exchange rates (3% in francs) to 60.5 billion francs ($66.9 billion). 30 January 2025
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for priority review a supplemental new drug application (sNDA) for Welireg (belzutifan), Merck & Co’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor. 28 January 2025
Biotechnology
Privately-held Swiss biotech AB2 Bio has entered into an option and licensing agreement with Japan’s Nippon Shinyak, which it describes as having extensive experience in marketing rare disease therapeutics in the USA. 27 January 2025
Biotechnology
Swiss pharma giant Roche today announced positive top-line results from year two of the EMBARK trial, a global, randomized, double-blind Phase III study of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy. 27 January 2025
Pharmaceutical
A final report from the Institute for Clinical and Economic Review (ICER) finds that the pricing of new therapies for the treatment of hereditary transthyretin amyloidosis (hATTR) “far exceeds commonly cited thresholds for cost effectiveness.” 8 October 2018
Biotechnology
Shares of US biotech firm Akcea Therapeutics gained 4.39% to $31.85 by close of trading and a further 2.04% to $32.50 in after-hours on Friday, after the company revealed that the US Food and Drug Administration had approved Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin (hATTR) amyloidosis in adults. 8 October 2018
Biotechnology
US biotech bluebird bio has had its marketing authorization application accepted by the European Medicines Agency for its investigational LentiGlobin gene therapy for treatment of adults and adolescents with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype. 5 October 2018
Biotechnology
UK-based biotech firm Orchard Therapeutics has been awarded the fast track PRIME Designation from the European regulator for its investigational gene therapy OTL-300. 4 October 2018
Biotechnology
Danish biotech firm Orphazyme today announced encouraging top-line results for its clinical Phase II/III trial with orally administered arimoclomol for the treatment of patients with Niemann-Pick disease Type C (NPC). 1 October 2018
Pharmaceutical
New Zealand’s Pharmaceutical Management Agency PHARMAC has announced a decision to list sapropterin (trade name Kuvan), a treatment for the rare condition phenylketonuria (PKU), for women who are pregnant or actively planning a pregnancy and require pharmacological support to manage PKU in pregnancy. 1 October 2018
Biotechnology
In an expert view, Maggie Chang, vice president, regulatory affairs, at China-based Hangzhou Tigermed Consulting, takes a look at what is now happening in China’s amended new drug approval system. 28 September 2018
Pharmaceutical
East Coast, USA-based Alnylam Pharmaceuticals has released positive topline results from an interim analysis of the ENVISION Phase III Study of givosiran. 28 September 2018
Biotechnology
It’s not often that the company announcing an acquisition sees a significant share price spurt, but this was certainly the case on Wednesday for Alexion Pharmaceuticals, which gained over 5.1% to $135.61. 27 September 2018
Biotechnology
Germany’s Boehringer Ingelheim yesterday announced that the Phase III LUME-Meso trial did not meet its primary endpoint of progression-free survival (PFS) [NCT01907100]. 26 September 2018
Biotechnology
Alexion Pharmaceuticals is in a strong position to push for a new approval with Soliris (eculizumab) after strong data in a rare complement-mediated disorder of the central nervous system. 25 September 2018
Biotechnology
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) at its September 2018 meeting recommended 13 medicines for approval, including three orphan medicines, with the latter being the following. 21 September 2018
Pharmaceutical
In what was clearly an active session for the Japanese Ministry of Health, Labor and Welfare (MHLW), four new drug approvals were announced this morning. 21 September 2018
Biotechnology
Following US approval last month, soon-to-be Takeda Pharmaceutical acquisition Shire has now also been granted authorization in Canada for Takhzyro (lanadelumab), as a treatment for hereditary angioedema (HAE). 20 September 2018
Biotechnology
Netherlands-based Pharming Group saw its share plunge 29.05% to 0.69 euros by mid-morning today, after it revealed a further setback in its attempt to widen the use of its leading drug. 19 September 2018
Biotechnology
Privately-held US biopharma firm Samumed has entered an exclusive license agreement for North American rights to its SM04646, being developed for the treatment of idiopathic pulmonary fibrosis (IPF). 17 September 2018
Pharmaceutical
Welcome to The Pharma Letter’s new column, RARE: Illuminating Orphan Diseases. Each column will take a deep dive into a rare disease – its cause, epidemiology, and treatment. Key questions will be answered: What is pharma doing for this disease? What still needs to be done? Are there any innovative treatments in the pipeline? We want to give rare disease patients a voice by shining a light on their condition and highlighting where further investment might be needed. 17 September 2018
Biotechnology
Approval of the immunotoxin Lumoxiti (moxetumomab pasudotox-tdfk), a first-in-class medicine for hairy cell leukemia, marks first new treatment option for patients in over 20 years 14 September 2018
Biotechnology
US biotech Amicus Therapeutics suffered a second setback in its bid for speedy approval for its Pompe diseases candidate, with the US Food and Drug Administration being negative following similar action from European regulators in June. 11 September 2018
Biotechnology
USA-based pharma firm Akcea Therapeutics (Nasdaq: AKCA) has said it will slash around 10% of its workforce in order to “better align with the immediate needs of its business”. 10 September 2018
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