UCB Brings First Therapy for Rare Disease TK2d to EU
UCB’s Kygevi, a combination of doxecitine and doxribtimine, received its first EU approval for treating thymidine kinase 2 deficiency (TK2d) under exceptional circumstances. The drug, aimed at patients whose disease began before age 12, cut mortality risk by 95% compared with historical untreated cohorts. Clinical data showed 84% of treated patients regained at least one motor function and 22% were able to discontinue respiratory support. The approval follows a similar FDA clearance in the United States and adds a rare‑disease therapy to the European market.
AZ Considers Filings for Long-Acting Strensiq Follow-Up
AstraZeneca’s Alexion unit intends to file a new long‑acting enzyme replacement therapy, efzimfotase alfa, as a successor to its Strensiq (asfotase alfa) for hypophosphatasia (HPP). Phase 3 data showed the drug met primary endpoints in pediatric patients but failed to achieve...
Blackstone Closes Record $6.3bn Life Sciences Fund
Blackstone announced that its sixth life sciences fund, BXLS VI, closed at $6.3 billion, making it the largest private fund ever raised for the sector. The fund was oversubscribed and is about 40% larger than the $4.6 billion fifth fund closed in 2020,...
Recordati Gets €10.9bn Takeover Bid From Private Equity Firm
Italian pharma group Recordati has received a €10.9 billion (≈ $12.5 billion) unsolicited takeover proposal from private‑equity firm CVC Capital Partners, which would take the company private and delist it from the Milan exchange. CVC already controls roughly 47% of Recordati’s shares, having...
Anavex Sinks After Pulling Alzheimer's Filing in EU
Anavex Life Sciences withdrew its European marketing authorisation application for blarcamesine, an add‑on therapy for early‑stage Alzheimer’s, after the EMA’s CHMP signaled a likely negative opinion. The committee criticized the trial’s efficacy data, methodological flaws, and safety reporting, including concerns...
Appeal Finds NICE Must Reconsider Alzheimer's Drugs
NICE has agreed to revisit its June 2025 rejection of Eli Lilly’s Kisunla (donanemab) and Eisai’s Leqembi (lecanemab) for NHS reimbursement. The appeal will send the dossiers back to the appraisal committee to re‑examine clinical benefits, long‑term data, infusion costs and unpaid...
First MHRA/NICE Aligned Guidance Due in June
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the National Institute for Health and Care Excellence (NICE) will begin delivering aligned guidance in June, enabling simultaneous licensing and reimbursement decisions for new medicines. Twenty‑seven companies have signed up...
Hope Rises for Vaccine Against Hookworm Parasite
A phase 2 trial of the Na‑GST1/Al–CpG vaccine demonstrated near‑complete protection against hookworm infection in healthy adults, with vaccinated participants shedding a median of zero eggs per gram versus 67 in the placebo group. The study, conducted in Washington, DC,...
US Court Says Pharma Giants Must Face False Claims Suit
The U.S. Ninth Circuit Court of Appeals denied dismissal motions by AbbVie, AstraZeneca, Novartis and Sanofi, allowing a whistleblower False Claims Act suit over alleged 340B program overcharges to proceed. The lawsuit, filed by Adventist Health System/West, claims the manufacturers...
Immutep Investors Spooked by LAG-3 Failure in Lung Cancer
Australian biotech Immutep saw its ASX shares tumble more than 88% after the independent data monitoring committee recommended halting its pivotal phase‑3 TACTI‑004 trial of the LAG‑3 inhibitor eftilagimod alfa (efti) in first‑line non‑small cell lung cancer. The trial, which...
Evotec Slashes Staff as Turnaround Plan Gathers Pace
Evotec unveiled the second phase of its Horizon turnaround, announcing 800 job cuts and the closure of four sites to save €75 million by 2027. The move follows a previous round of 600 layoffs and a failed $2.1 billion Halozyme takeover, reducing...
Lundbeck Names AI Head in Quest to Become 'Bionic' Company
Lundbeck has promoted Markus Kede to chief AI officer, shifting him from finance to lead the drugmaker's AI agenda. The newly created role reports directly to CEO Charl van Zyl and will be based in Sweden. Kede will oversee AI...
Roche's Oral SERD Flunks Phase 3 Breast Cancer Test
Roche’s oral SERD giredestrant failed to meet its primary endpoint in the phase 3 persevERA trial, showing no significant improvement over letrozole when combined with palbociclib in first‑line HR‑positive, HER2‑negative advanced breast cancer. The company now limits U.S. filing to ESR1‑mutated...
Safety Concerns Prompt Ipsen to Pull Tazverik From Market
Ipsen is voluntarily withdrawing its EZH2 inhibitor Tazverik worldwide after an interim safety analysis in the phase 1b/3 SYMPHONY‑1 trial linked the drug to secondary hematologic malignancies. The pull‑back includes terminating all ongoing Tazverik studies and recalling the product in markets...
Can GLP-1 Drugs Help Tackle Addiction?
A new BMJ meta‑analysis of over 600,000 U.S. veterans with type‑2 diabetes found that patients prescribed GLP‑1 agonists such as semaglutide or tirzepatide were 14% less likely to develop substance‑use disorders (SUD) than those on SGLT2 inhibitors. Over a three‑year...
Hansa Closes on FDA Verdict for Transplant Drug Imlifidase
The FDA has begun reviewing Hansa Biopharma’s imlifidase, a drug that desensitises highly sensitised kidney‑transplant patients, with a decision expected by December 19, 2024. If approved, it would be the first U.S. therapy to improve transplant odds for the 10‑15 % of...
Five Projects Share £100m UK-US Cancer Grand Challenges Fund
Five interdisciplinary teams will receive up to £20 million each from the UK‑US Cancer Grand Challenges, adding £100 million to the programme and raising total investment to £465 million since 2016. The five projects, spanning 34 institutions in nine countries, target unconventional angles...
UK Puts £50m Behind Expanded Clinical Trials Drive
The UK government announced nearly £48 million in equipment funding to accelerate commercial clinical trials across the NHS. The money will equip 51 NHS trusts and 79 primary‑care organisations with diagnostic tools, scanners and mobile research vans, with 60 % directed to...
'Dismal' Survival Demands Change in Bile Duct Cancer Therapy
An international panel of 147 oncology leaders, including Prof. John Bridgewater, issued a consensus calling for sweeping reforms in the diagnosis, treatment, and research of cholangiocarcinoma (bile duct cancer). The disease remains deadly, with three‑quarters of patients dying within a...
Quell Takes New Treg Into Clinic After Transplant Study Halt
Quell Therapeutics has launched the phase 1/2 CHILL trial of its autologous CAR‑Treg therapy QEL‑005 in rheumatoid arthritis and systemic sclerosis, after pausing the liver‑transplant program QEL‑001. The multi‑center study will enroll patients in the UK, Germany and Spain, with read‑out...
CMS Extends Deadline for MFN Pricing Pilot Scheme
The U.S. Centers for Medicare & Medicaid Services (CMS) has pushed back the deadline for manufacturers to join its GENEROUS pilot, extending the initial cut‑off to April 30 while keeping the final deadline at June 30. The voluntary program applies a Most‑Favoured...
NHS "Isn't Delivering Equitable Care for Rare Diseases"
Genetic Alliance UK’s equity report warns the NHS is falling short for the 3.5 million Britons living with rare diseases. A quarter of patients wait three years for a diagnosis, only 5 % of conditions have approved treatments, and just 10 % of...
Boehringer Bets up to $500m on Sitryx Immunology Programme
Boehringer Ingelheim announced a potential $500 million investment in UK biotech Sitryx to obtain global rights to an undisclosed small‑molecule immunology programme. The partnership covers upfront fees, milestones and royalties, giving Boehringer full responsibility for research, clinical development and commercialization. Sitryx’s...
Asahi Kasei Buys Antiviral Firm Aicuris for €780m
Japanese conglomerate Asahi Kasei announced a €780 million acquisition of German biotech Aicuris, gaining access to the oral antiviral pritelivir for herpes simplex virus (HSV). The drug, poised for FDA approval later this year, has shown superior lesion healing in phase‑3...
In Brief SOTU Remarks, Trump Asks Congress to "Codify" MFN
President Donald Trump used a brief four‑minute segment of his State of the Union address to push Congress to codify his Most‑Favoured Nations (MFN) drug‑pricing program. He claimed the voluntary agreements would give Americans the lowest prescription prices worldwide, though...
FDA Starts Review of Regeneron's Drug for Rare Disease FOP
The FDA has placed Regeneron's anti‑activin A antibody garetosmab under priority review, with a decision expected by August. Phase 3 OPTIMA data showed a 94% reduction in new heterotopic ossification lesions at the lower dose and a 90% reduction at the higher...
UK Firm Steps in to Ease Medicine Supply Crisis in NHS
A shortage of bone cement from German supplier Heraeus Medical threatens NHS orthopaedic surgeries, potentially lasting two months. NHS England has asked trusts to prioritise emergency joint replacements, leaving elective patients at risk. UK‑based Biocomposites accelerated the launch of its...
Ex-Novartis CMO John Tsai Joins Daiichi Sankyo
Daiichi Sankyo appointed former Novartis chief medical officer John Tsai as its new CMO, succeeding Ken Takeshita after a five‑year tenure. Tsai brings a record of launching 160 projects and 15 regulatory approvals, including Zolgensma and Pluvicto, and recent experience leading biotech...
Novartis' Oral BTK Drug Moves the Needle in CINDU
Novartis announced that its oral BTK inhibitor Rhapsido (remibrutinib) achieved significantly higher complete response rates than placebo in the phase 3 RemIND trial for the three most common forms of chronic inducible urticaria (CINDU). The drug, already approved for chronic spontaneous...
NICE Backs First Disease-Modifying Drug for ARG1 Deficiency
Immedica’s pegzilarginase (Loargys), the first enzyme replacement therapy for arginase‑1 (ARG1) deficiency, has received NICE endorsement for NHS use in the UK. The weekly IV or subcutaneous treatment cuts blood arginine levels by roughly 80% and is recommended for patients...
Roche Trial Offers Hope to Patients with Rare Kidney Disease
Roche announced that its anti‑CD20 antibody Gazyva (obinutuzumab) met the primary endpoint in the phase 3 MAJESTY trial for primary membranous nephropathy (pMN). The study showed significantly higher complete remission rates at two years compared with the immunosuppressant tacrolimus, while maintaining...
Bankruptcy Threat Looms as Quince Runs Out of Options
Quince Therapeutics warned investors it may need bankruptcy protection after its lead drug eDSP failed a pivotal phase 3 trial for ataxia‑telangiectasia. The SEC filing disclosed less than $6 million in cash, $12 million in short‑term investments and a $16 million unsecured line of...
PTC Pulls File for Duchenne Therapy on FDA Feedback
PTC Therapeutics has withdrawn its FDA filing for Translarna (ataluren) as a treatment for nonsense‑mutation Duchenne muscular dystrophy after the agency indicated the data would not meet the threshold for substantial evidence of effectiveness. The decision ends a decade‑long effort...
FDA Kicks Off Review of Takeda's Narcolepsy Hopeful
The U.S. Food and Drug Administration has placed Takeda's orexin‑2 receptor agonist oveporexton (TAK‑861) into a priority review, with a decision expected in the third quarter. If approved, it would become the first drug to target the underlying orexin deficiency...
Lupin Settles Myrbetriq Patent Spat with Astellas for $90m
Lupin has agreed to pay $90 million to settle U.S. patent litigation with Astellas over the over‑active bladder drug Myrbetriq. The settlement includes a $75 million option payment and ongoing per‑unit license fees until Astellas' patent expires in September 2027. The deal lets...
Data Builds on Roche's Dominance in Primary Progressive MS
Roche disclosed phase 3 data showing its oral BTK inhibitor fenebrutinib is non‑inferior to Ocrevus in primary progressive multiple sclerosis (PPMS). The FENtrepid trial demonstrated a 12% relative reduction in disability progression risk over 24 weeks, with a notable 26% improvement...
Novartis Breaks Ground on San Diego Biomedical R&D Site
Novartis has broken ground on a $1.1 billion, 466,000‑sq‑ft biomedical research centre in San Diego, the flagship of a $23 billion U.S. capital investment programme. The campus, slated to open around 2029, will house roughly 1,000 scientists and integrate the company’s West...
Leqembi Starts to Deliver for Eisai and Biogen
Biogen and Eisai reported a 54% jump in fourth‑quarter sales of their Alzheimer’s drug Leqembi, reaching $134 million, with $78 million generated in the United States. The increase follows the FDA’s approval of a once‑weekly subcutaneous injection, offering a less‑burdensome alternative to...
Hims & Hers Copies Novo Nordisk's New Wegovy Pill
Telehealth firm Hims & Hers has launched an oral semaglutide pill priced at $49 per month, directly copying Novo Nordisk’s newly released oral Wegovy that sells for $149. Novo reports strong uptake of its product, with roughly 50,000 prescriptions filled...
Grail Multi-Cancer Test Taps Into Hims & Hers Network
Grail has partnered with telehealth firm Hims & Hers to sell its Galleri multi‑cancer blood test at a $250 discount off the $949 list price. The collaboration follows Grail’s recent filing for FDA pre‑market approval, based on interim results from...
Abbott's Wireless Heart Failure Sensor Cleared for NHS Use
Abbott’s CardioMEMS HF wireless sensor has received NICE approval for routine NHS use in chronic heart‑failure patients. The tiny implant measures pulmonary artery pressure and transmits daily readings via a pillow‑embedded antenna, allowing clinicians to adjust medication remotely. Clinical trials...
GSK Delivers in New CEO Miels' First Financial Update
Luke Miels’ first financial update as GSK chief executive shows an 8% rise in fourth‑quarter sales to £8.62 bn, lifting full‑year revenue 7% to £32.67 bn. Growth was driven by new launches such as Blenrep, Nucala and Exdensur, and a pipeline bolstered...
Patent Losses Hit Novartis Hard, but CEO Has a Plan
Novartis faced its largest patent cliff in Q4 2025, losing exclusivity on Entresto, Tasigna and Promacta/Revolade, which dragged quarterly sales down 1% to $13.3 billion. CEO Vas Narasimhan projects a $4 billion sales hit in 2026 but expects mid‑term guidance to stay on track,...
Fears US Drug Pricing Deal Will Weigh Heavy on the NHS
UK Science Minister Sir Patrick Vallance confirmed that the £1 billion three‑year drug pricing deal with the United States will be funded from the Department of Health and Social Care budget, not the Treasury. The agreement keeps US tariffs on UK‑origin...
FDA Knocks Back AstraZeneca's Self-Injected Lupus Drug
AstraZeneca's subcutaneous formulation of its lupus biologic Saphnelo was rejected by the FDA, receiving a complete response letter despite recent European approval. The IV version continues to grow, posting $483 million in sales for the first nine months of 2025, and...
UK Will Cover Transport Costs for Children with Cancer
The UK government will allocate a £10 million fund to cover transport costs for children and young people up to age 24 diagnosed with cancer, with the scheme rolling out by 2027. The support is universal, irrespective of household income, and...
UK Body Raps Sanofi for Disparaging Pfizer's RSV Jab
Sanofi was found to have breached the UK pharmaceutical code after its chief executive, Paul Hudson, made unbalanced statements in an Observer interview comparing the company’s RSV antibody Beyfortus with Pfizer’s vaccine Abrysvo. The Prescription Medicines Code of Practice Authority...
FDA Knocks Back Pharming's Bid for Wider Joenja Use
The U.S. Food and Drug Administration issued a complete response letter rejecting Pharming's request to extend Joenja (leniolisib) to children aged four to 11 with activated phosphoinositide 3‑kinase delta syndrome (APDS). The agency flagged potential under‑exposure in lower‑weight pediatric patients...
Mixed Data Mars Sanofi's Venglustat Programme
Sanofi announced that its oral glucosylceramide synthase inhibitor venglustat achieved statistically significant neurological improvements in a Phase 3 trial for type 3 Gaucher disease, prompting regulatory filing plans. The same drug failed to demonstrate efficacy in a Fabry disease study, adding to...
FDA Launches Pilot Programme to Boost US Manufacturing
The FDA has opened its PreCheck pilot, a two‑phase program designed to speed regulatory approval for new pharmaceutical manufacturing sites in the United States. The agency will select facilities this year based on national‑priority products, development stage, and timeline to...
