As stakeholders in the pharmaceutical industry, we all have a responsibility to ensure life-saving treatments promptly and safely reach patients regardless of where they live. To this end, by integrating early access programs and other less conventional strategies into a company’s business model for drug distribution, patients who are suffering from life-threatening conditions can gain access to needed treatments in regions where there is a lack of drug accessibility, while at the same time offering benefits to pharmaceutical companies. 2 October 2024
Venclexta (venetoclax), in combination with azacytidine, has been granted Breakthrough Therapy Designation (BTD) by the US Food and Drug Administration (FDA) for previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International prognostic scoring system (IPSS-R). 21 July 2021
National Health Service (NHS) patients in England are set to benefit from early access to potentially life-saving new medicines, including cutting-edge gene therapies, thanks to a new Innovative Medicines Fund and £680 million ($925 million) of ringfenced funding, the NHS chief executive has announced today. 21 July 2021
The US Food & Drug Administration approval of Bylvay (odevixibat), the first drug approved for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC), developed by Boston, USA-based Albireo Pharma. 21 July 2021
The European Commission (EC) has approved the marketing authorization application for Ryeqo (relugolix 40mg, estradiol 1.0mg, and norethindrone acetate 0.5mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age, with no limitation for duration of use. 21 July 2021
The US Food and Drug Administration has approved fexinidazole as the first all-oral treatment for both stages of the Trypanosoma brucei gambiense form of sleeping sickness (Human African trypanosomiasis) in patients six years of age and older and weighing at least 20 kg. 21 July 2021
The European Medicines Agency’s human medicines committee (CHMP) has started a rolling review of Vidprevtyn, a COVID-19 vaccine developed by Sanofi Pasteur. 20 July 2021
Swiss pharma giant Roche today announced that Japan’s Ministry of Health Labor and Welfare (MHLW) has approved Ronapreve (casirivimab and imdevimab), for the indication of SARS-CoV-2 infection in patients with mild to moderate COVID-19 via intravenous infusion. 20 July 2021
Shares of California, USA-based cardiorenal specialist Ardelyx plunged more than 70% to $2.28 in after-hours trading on Monday, as the company revealed the US regulator is not ready to approve its tenapanor for dialysis patients. 20 July 2021
In anticipation of the third wave of the pandemic, the Russian government has significantly increased purchases of anti-COVID-19 drugs, according to recent statements by state press-service and local media reports. 20 July 2021
Drug prices are steadily rising in Russia due to the weak rouble and the ever-growing prices for raw materials, most of which are traditionally imported to Russia from abroad, reports The Pharma Letter’s local correspondent. 20 July 2021
The US Food and Drug Administration (FDA) has granted Priority Review designation for the Biologics License Application (BLA) for the COVID-19 vaccine from Pfizer and BioNTech. 19 July 2021
The European Medicines agency announced today that it has started evaluating an application to extend the use of Kineret (anakinra) to include treatment of coronavirus disease 2019 (COVID-19) in adult patients with pneumonia who are at risk of developing severe respiratory failure (inability of the lungs to work properly). 19 July 2021
Imfinzi (durvalumab) has been approved in China for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC), in combination with standard of care platinum chemotherapy (etoposide plus a choice of either carboplatin or cisplatin). 19 July 2021
The US Food and Drug Administration (FDA) approved Vaxneuvance (pneumococcal 15-valent conjugate vaccine) for active immunization for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F in adults 18 years of age and older. 19 July 2021
Licensing agreements featured heavily in last week’s news, starting with US biotech Biogen entering into an agreement that could cost it $1 billion to get rights to a BTK inhibitor – orelabrutinib for multiple sclerosis – from China’s InnoCare Pharma. Denmark’s Novo Nordisk in-licensed rights to Prothena’s ATTR amyloidosis program, and PRX004, in a deal worth up to $1.2 billion. Also, Ionis Pharmaceuticals last Tuesday exercised an option on fellow USA-based Bicycle Therapeutics’ oligonucleotide targeting the transferrin receptor 1. On the M&A front, Eli Lilly revealed on Wednesday it was acquiring privately-held US company Protomer Technologies for what could be a $1 billion transaction, getting rights to its glucose-sensing insulin project. Additionally, the US Food and Drug Administration took issue with Amgen’s promotion of its cancer drug Neulasta, which the agency said could undermine confidence in biosimilars. 18 July 2021
Shares of USA-based Kadmon Holdings shot up more than 20% to $4.28 on Friday, after the company revealed its pharma unit had received its first regulatory approval, for Rezurock (belumosudil). 17 July 2021
The US Food and Drug Administration on Friday approved a new use for Prograf (tacrolimus) based on a non-interventional (observational) study providing real-world evidence (RWE) of effectiveness. 17 July 2021
The European Medicines Agency has validated and accepted the marketing authorization application (MAA) for surufatinib for the treatment of pancreatic and extra-pancreatic (non-pancreatic) neuroendocrine tumors (NETs), submitted by China’s Hutchmed. 16 July 2021
USA-based FibroGen's shares have dropped by more than a third after a scientific panel of the US Food and Drug Administration voted against approval of roxadustat. 16 July 2021
Looking to overturn a regulatory rebuff, New Jersey, USA’s Merck & Co has announced positive event-free survival (EFS) data from the pivotal neoadjuvant/adjuvant Phase III study KEYNOTE-522. 16 July 2021
UK-based ViiV Healthcare, the specialist HIV company majority-owned by GSK (LSE: GSK), with Pfizer and Shionogi as shareholders, has announced a milestone in its efforts to achieve widespread international access to its therapies. 7 October 2024
Texas Attorney General Ken Paxton has filed a lawsuit against major pharmaceutical companies, including Eli Lilly (NYSE: LLY), alleging a conspiracy to artificially inflate the prices of insulin by up to 1,000%. 7 October 2024
Kezar Life Sciences has paused its Phase IIb PALIZADE trial evaluating zetomipzomib in lupus nephritis (LN) following the deaths of four patients. 7 October 2024
India's new Biotechnology Research Innovation and Entrepreneurship Development (Bio-RIDE) scheme is a significant step towards consolidating and advancing the country's biotechnology initiative. 7 October 2024
The Irish Pharmaceutical Healthcare Association (IPHA), the representative body for the research-based biopharmaceutical industry in Ireland, says it welcomes the allocation of 30 million euros ($32.9 million) for new medicines in Budget 2025. 7 October 2024
Important regulatory news last week included French pharma major Sanofi gaining approval from the US Food and Drug Administration (FDA) and the Chinese medicines authority for its blockbuster drug Dupixent as the first biologic for chronic obstructive pulmonary disease (COPD). Spanish epigenetics firm Oryzon Genomics announced that, with FDA feedback, it is preparing for a Phase III trial of its CNS drug candidate vafidemstat. Also, the FDA authorized Bristol Myers Squibb’s blockbuster drug Opdivo in a new lung cancer indication for patients with specific mutations such as EGFR or ALK. On the deal-making front, US biotech Arcus Biosciences has entered a collaboration with AstraZeneca to evaluate its casdatifan with the UK pharma major’s volrustomig in renal cell cancer. 6 October 2024
The European Medicines Agency’s pharmacovigilance committee, PRAC, has started a review of medicines containing finasteride and dutasteride following concerns regarding suicidal ideation (suicidal thoughts) and behaviors. 4 October 2024
Proposals from the National Health Service (NHS) England have been set out for a phased launch of Eli Lilly’s obesity injection Mounjaro/Zepbound (tirzepatide) at the start of a consultation. 4 October 2024
Bristol Myers Squibb has received a new approval from the US Food and Drug Administration for its blockbuster checkpoint blocker, Opdivo (nivolumab). 4 October 2024
The US Food and Drug Administration (FDA) has removed the partial clinical hold on delpacibart etedesiran (del-desiran/AOC 1001), an investigational treatment designed to address the root cause of myotonic dystrophy type 1 (DM1), under development by US biotech Avidity Biosciences . 4 October 2024
The US Food and Drug Administration (FDA) has granted the Gilead Sciences subsidiary Kite a Regenerative Medicine Advanced Therapy Designation (RMAT) for Yescarta (axicabtagene ciloleucel) for adults with newly-diagnosed, high-risk large B-cell lymphoma (LBCL). 3 October 2024
The US Food and Drug Administration (FDA) has determined the shortage of tirzepatide injection, a glucagon-like peptide 1 (GLP-1) medication, has been resolved. 3 October 2024
AstraZeneca’s supplemental New Drug Application (sNDA) for Calquence (acalabrutinib) has been accepted and granted Priority Review in the USA for the treatment of adults with previously untreated mantle cell lymphoma (MCL). 3 October 2024
The US Department of Health and Human Services (HHS) has released final guidance for the second cycle of Medicare drug price negotiations. 3 October 2024
The European Medicines Agency (EMA) and the European medicines regulatory network are working to speed up the approval of new medicines. 3 October 2024
Spanish epigenetics firm Oryzon Genomics has announced the receipt of the official meeting minutes from a recent End-of-Phase II meeting for vafidemstat in borderline personality disorder (BPD) with the US Food and Drug Administration (FDA). 2 October 2024
As stakeholders in the pharmaceutical industry, we all have a responsibility to ensure life-saving treatments promptly and safely reach patients regardless of where they live. To this end, by integrating early access programs and other less conventional strategies into a company’s business model for drug distribution, patients who are suffering from life-threatening conditions can gain access to needed treatments in regions where there is a lack of drug accessibility, while at the same time offering benefits to pharmaceutical companies. 2 October 2024
The US Food and Drug Administration (FDA) yesterday released its fiscal year 2023 (FY23) Generic Drug User Fee Amendments (GDUFA) Science and Research Outcomes Report. 2 October 2024
CSL Behring has been granted a positive recommendation by the Spanish Interministerial Commission on the Pricing of Medicines (CIPM), resulting in national reimbursement for Hemgenix (etranacogene dezaparvovec) for eligible haemophilia B patients in Spain. 1 October 2024