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Biotechnology
Shares in Californian biotech Rigel Pharmaceuticals (Nasdaq:RIGL) leapt a tenth yesterday on news that the US regulator had approved Tavalisse (fostamatinib disodium hexahydrate) to treat adult patients with chronic immune thrombocytopenia (ITP). 18 April 2018
Biotechnology
The US Food and Drug Administration has approved Crysvita (burosumab), the first drug cleared to treat adults and children ages 1 year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets. 18 April 2018
Biotechnology
UK-based cannabinoid specialist GW Pharmaceuticals was up by a double-digit percentage by Tuesday afternoon as investors looked at the latest news on its New Drug Application for Epidiolex (cannabidiol or CBD). 17 April 2018
Biotechnology
Analysts at Jeffries have predicted that Roche’s Hemlibra (emicizumab) could generate $5 billion in annual sales – provided its use for hemophilia A is widened to the larger, non-inhibitor population. 17 April 2018
Biotechnology
The combination of Opdivo (nivolumab) 3mg/kg plus Yervoy (ipilimumab) 1mg/kg (injections for intravenous use) has received priority approval from the US Food and Drug Administration as the first immuno-oncology combination therapy for previously untreated patients with intermediate- and poor-risk advanced renal cell carcinoma (RCC). 17 April 2018
Biotechnology
It is not uncommon for drugmakers to imply that mistaken regulatory staff or a misunderstanding are the reason for development setbacks. 16 April 2018
Biotechnology
The US Food and Drug Administration has lifted the clinical hold on studies of BPX-501 in the USA. 14 April 2018
Biotechnology
Massachusetts, USA-based messenger RNA (mRNA) specialist Translate Bio says the US Food and Drug Administration has cleared the company to begin a first-in-human clinical trial of MRT5005 in patients with cystic fibrosis. 13 April 2018
Generics
The European Medicines Agency and its European and international partners have successfully strengthened their interactions to improve the oversight of active pharmaceutical ingredient (API) manufacturers worldwide, as highlighted in the International API inspection program report for 2011-2016, published today. 12 April 2018
Pharmaceutical
More drug approvals, a busy US Food and Drug Administration (FDA) and the growing success of small and medium sized pharma companies are some of the major findings of a comparison published on Thursday. 12 April 2018
Biotechnology
The US Food and Drug Administration has said that it requires extended time to review additional information regarding the results of liver function tests provided in connection with its New Drug Application (NDA) for elagolix in endometriosis-associated pain. 12 April 2018
Biosimilars
During the recent National Health Policy Conference run by America’s Health Insurance Plans, the US Food and Drug Administration’s Commissioner Dr Scott Gottlieb shared his candid remarks on the state of the pharmaceutical market in the USA.1 While drug prices are traditionally not part of the FDA’s responsibilities, the physician and cancer survivor was passionate about highlighting his observations of the American healthcare industry, says Dr Nicola Davies in her monthly exclusive blog for The Pharma Letter. 12 April 2018
Pharmaceutical
The US Food and Drug Administration has approved Afinitor Disperz (everolimus tablets for oral suspension), for the adjunctive treatment of adult and pediatric patients aged two years and older with tuberous sclerosis complex (TSC)-associated partial-onset seizures. 11 April 2018
Biotechnology
Shares in Verastem were lifted about 10% yesterday as the East Coast, USA-based biotech firm revealed that the US drugs regulator is to assess its oncology candidate duvelisib under the priority review scheme. 10 April 2018
Pharmaceutical
On the bad news front last week, USA-based Alkermes received a refuse-to file letter from the US Food and Drug Administration for its depression candidate ALKS-5461. Attracting attention on the deal-making scene was Germany’s Boehringer Ingelheim immuno-oncology collaboration with OSE Immunotherapeutics, and Switzerland’s Ferring agreeing to acquire microbiome specialist Rebiotix. Research news highlighted two failures. First, the disappointment of Phase III date on GenSight’s rare disease drug candidate GSO10, and then, more spectacular, the failure of Incyte Corp and Merck & Co’s combination trial of Keytruda and epacadostat in melanoma. 8 April 2018
Pharmaceutical
Shares of Clovis Oncology gained 5.85% to $55.52 on the news that its PARP inhibitor Rubraca (rucaparib) had won US approval for an additional indication under priority review. 7 April 2018
Biosimilars
Following warnings from the US Food and Drug Administration last year, and again in February, related to its manufacturing practices, South Korean drugmaker Celltrion has now been handed a Complete Response Letter from the agency related to two of its proposed biosimilars. 6 April 2018
Pharmaceutical
Dacomitinib took a step towards becoming a new treatment option in locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC) on Wednesday. 5 April 2018
Pharmaceutical
The European Medicines Agency (EMA) has accepted for review two applications from French drugmaker Sanofi, the firm revealed today. 3 April 2018
Pharmaceutical
Shares in Ireland-headquartered firm Alkermes closed 22% down on Monday at $45.23, a market reaction prompted by the US Food and Drug Administration’s (FDA) Refusal to File letter relating to the New Drug Application (NDA) for a proposed depression treatment. 3 April 2018
Pharmaceutical
The nomination of Robert F Kennedy Jr — known as RFK Jr — for the role of US Secretary of the Department of Health and Human Services (HHS) President-elect Donald Trump sent shares in vaccine companies sharply downwards. 22 November 2024
Biotechnology
British pharma major AstraZeneca is facing challenges in its bid to secure full approval for its anticoagulant reversal therapy, Andexxa (andexanet alfa), in the USA. 22 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval of Ziihera (zanidatamab-hrii) 50mg/mL for injection for intravenous use, say the drug’s developer, Jazz Pharmaceuticals. 21 November 2024
Biotechnology
Astellas Pharma has been hit by bad news from the US Food and Drug Administration, which will not currently approve the firm’s request to supplement the label for Izervay (avacincaptad pegol). 21 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has accepted for review the resubmission of the supplemental Biologics License Application (sBLA) for Dupixent(dupilumab), Regeneron and partner Sanofi revealed late Friday. 18 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Syndax Pharmaceuticals’ Revuforj (revumenib). 18 November 2024
Biotechnology
Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency. 15 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the treatment of AADC (aromatic L-amino acid decarboxylase) deficiency, making it the first-ever gene therapy approved in the USA that is directly administered to the brain. 14 November 2024
Biotechnology
Shares of US genetic meds company MeiraGTx Holdings were up 7% at $7.12 in early trading after it announced important developments along with its third-quarter 2024 financials, reporting a $0.54 per share loss. 13 November 2024
Pharmaceutical
Intercept Pharmaceuticals, now wholly owned subsidiary of Italy’s Alfasigma, today revealed that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) that addresses the supplemental New Drug Application (sNDA) for Ocaliva (obeticholic acid, OCA) seeking full approval for the treatment of indicated patients with primary biliary cholangitis (PBC) – a rare, progressive disease that disproportionally affects women. 12 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug (IND) application for its COVID-19-Influenza Combination (CIC) and stand-alone influenza vaccine candidates, US vaccines developer Novavax revealed yesterday. 12 November 2024
Biotechnology
RAPT Therapeutics has announced the termination of its zelnecirnon program following regulatory concerns around safety. 12 November 2024
Biotechnology
On November 8, the US Food and Drug Administration approved Aucatzyl (obecabtagene autoleucel; obe-cel) from US biopharma Autolus Therapeutics' 11 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program, according to Sarah Wicks and James Valentine at US law firm Hyman, Phelps & McNamara. 11 November 2024
Pharmaceutical
Shanghai-based Dizal has made a submission to the US Food and Drug Administration, seeking approval for sunvozertinib. 8 November 2024
Biotechnology
US healthcare giant Johnson & Johnson today revealed it has submitted applications with the US Food and drug Administration (FDA) and European Medicines Agency (EMA) to expand the approved indications for its Darzalex (daratumumab) subcutaneous (SC) formulation for the treatment of high-risk smouldering multiple myeloma (SMM). 8 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Pharmaceutical
USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024
article
The US Food and Drug Administration (FDA) has announced approval of a modification to the Opioid Analgesic Risk Evaluation and Mitigation Strategy (OA REMS) 6 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, under development by Ionis Pharmaceuticals. 4 November 2024
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