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
AstraZeneca’s planned $39 billion buy of American rare disease specialist Alexion Pharmaceuticals hit a speed bump on Tuesday, after the UK’s antitrust body launched a review of the deal. 26 May 2021
Shares in New Jersey, USA-based company Provention Bio rose by over a quarter on Tuesday, after the firm announced positive regulatory news related to its type 1 diabetes (T1D) candidate teplizumab. 26 May 2021
The European Commission (EC) should exempt advanced therapies from Genetically Modified Organism (GMO) legislation, which hurts Europe’s ability to attract clinical trials and delays patient access to transformative medicines. 26 May 2021
The USA’s Institute for Clinical and Economic Review (ICER) yesterday posted a Protocol outlining how it will conduct its first annual assessment into how well major insurers’ prescription drug coverage policies align with a set of fair access standards developed by ICER with expert input from patient advocates, clinician specialty societies, US payers, and life science companies. 26 May 2021
The US Food and Drug Administration has accepted the Biologics License Application (BLA) for ublituximab, an investigational glycoengineered anti-CD20 monoclonal antibody, in combination with Ukoniq (umbralisib), TG Therapeutics’ once-daily, oral, inhibitor of PI3K-delta and CK1-epsilon, as a treatment for patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). 26 May 2021
The European Commission has approved Venclyxto (venetoclax) in combination with hypomethylating agents, azacitidine and decitabine, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. 26 May 2021
Johnson & Johnson subsidiary Janssen has received approval from the European Commission for Ponvory (ponesimod) for the treatment of adult patients with relapsing multiple sclerosis (RMS) with active disease defined by clinical or imaging features. 25 May 2021
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Verastem’s investigational RAF/MEK inhibitor VS-6766, combined with defactinib, its FAK inhibitor, in recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy. 25 May 2021
New bipartisan legislation has been proposed by two US Representatives that would fund critical research on treatments and cures for diseases including Alzheimer’s, cancer, blindness, juvenile diabetes, and sickle-cell anemia amid research delays caused by the pandemic. 25 May 2021
The Russian government will use the mechanism of interventions in the domestic pharmaceutical market to tighten control of drug prices, according to recent statements by some senior state officials and local media, reports The Pharma Letter’s local correspondent. 25 May 2021
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) for Boehringer Ingelheim’s BI 425809 for the treatment of cognitive impairment associated with schizophrenia (CIAS). 24 May 2021
The US Food and Drug Administration strictly prohibits the distribution of counterfeit, diverted, subpotent, substandard, adulterated, misbranded, and expired drugs that pose a serious threat to life. Nevertheless, such drugs continue to end up in circulation, compromising patient safety. 24 May 2021
Last Saturday, US biotech Moderna announced that the Ministry of Food and Drug Safety of South Korea (MFDS) has approved the company’s application for conditional marketing authorization for its mRNA vaccine against COVID-19. 24 May 2021
On Friday, the US Food and Drug Administration approved Rybrevant (amivantamab-vmjw) as the first treatment for adult patients with non-small cell lung cancer (NSCLC) whose tumors have specific types of genetic mutations: epidermal growth factor receptor (EGFR) exon 20 insertion mutations. 22 May 2021
The European Medicines Agency’s Committee for Human Medicinal Products (CHMP) has issued a positive scientific opinion following the referral of sotrovimab to the CHMP under Article 5(3) of Regulation 726/2004. 21 May 2021
Following its May 2021 meetings, the European Medicines Agency’s human medicines committee (CHMP) recommended eight even medicines, including one gene therapy and one generic, for approval. 21 May 2021
New storage conditions for the COVID-19 vaccine from Pfizer and Germany’s BioNTech have today been approved by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). 21 May 2021
With the Olympic Games just a couple of months away and coronavirus cases in the country at a dangerous point, Japan has approved two vaccines in a single day. 21 May 2021
The US Food and Drug Administration has approved another important indication for Opdivo (nivolumab, injection for intravenous use), this one for the adjuvant treatment of completely resected esophageal or gastroesophageal junction (GEJ) cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT). 21 May 2021
US biotech Vertex Pharmaceuticals says that the European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) have validated the post-marketing applications for an expanded indication of Kaftrio. 20 May 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