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  • Regulatory NewsRegulatory News

    Novartis Admits to ‘Mistake’ After Partial Clinical Hold Placed on Zolgensma Trial

    Novartis on Wednesday said that the US Food and Drug Administration (FDA) placed a partial hold on intrathecal clinical trials of its gene therapy Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) patients based on findings in a small preclinical animal study.   On Thursday, a Novartis spokesperson told Focus that a draft report of the preclinical safety findings was presented to the AveXis safety management team last March and the company “...
  • ReconRecon

    Recon: Novartis Wins FDA Approval for Eye Drug; New ICER Report on Net Drug Price Hikes

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Novartis gets FDA boost in crowded eye drug market ( Reuters ) ( PMLive ) ( Press ) ICER Identifies Costliest US Drug-Price Hikes That Are Not Supported by New Clinical Evidence ( ICER ) ( Reuters ) ( Stat -$) Continuing Appropriations Act Changes Treatment of Authorized Generics in Medicaid Rebate Average Manufacturer Price ( FDA Law Blog ) FDA leaders worry ...
  • Regulatory NewsRegulatory News

    Novartis Blames Zolgensma Data Manipulation on Two Fired Senior Executives

    In its response to a US Food and Drug Administration (FDA) Form 483, Novartis said in a document released Tuesday that two senior executives at its subsidiary AveXis “could not offer a credible explanation for revisions to and inconsistencies in the data” and were later fired from their positions in August. The firings follow the revelation last month that AveXis manipulated some of the nonclinical data included in the biologic license application for its billion-dollar...
  • Regulatory NewsRegulatory News

    Novartis Inhaler is First Device to be Certified Under MDR

    BSI’s UK notified body announced Wednesday that the Novartis Concept1 inhaler is the first device to be certified under the new EU Medical Devices Regulation (MDR). While previously classified as a Class I device, meaning it did not need to be reviewed by a notified body, the inhaler is now a Class IIa device under Rule 20 of MDR, which requires the notified body review. Gary Slack, SVP of medical devices at BSI said:  “As a result of being the first designated EU N...
  • Regulatory NewsRegulatory News

    Novartis Makes New Data Integrity Commitment

    Looking to slow the fallout from Novartis’ data manipulation fiasco with its billion-dollar gene therapy Zolgensma (onasemnogene abeparvovec-xioi), CEO Vasant Narasimhan told an investor conference on Monday that the company will be more proactive in reporting data integrity issues to the US Food and Drug Administration (FDA). “We are voluntarily and proactively taking a pledge with the FDA to ensure that we will inform them within five business days of any credible all...
  • Regulatory NewsRegulatory News

    Do People Understand Cancer Drug Ads on TV? Drugmakers Weigh in on FDA Research

    As television ads for cancer drugs pose the question, “Who wouldn’t want a chance to live longer?” or present statements like: “Living longer is possible,” the US Food and Drug Administration’s (FDA) Office of Prescription Drug Promotion (OPDP) is trying to figure out if consumers can understand these drugs’ specific and often complicated indications supported by different types of endpoints. And now drugmakers Novartis, Merck and Eli Lilly are seeking modifications to ...
  • Regulatory NewsRegulatory News

    Questions Pile up for Novartis as Senators Call on FDA to Take Action

    Why wait three months to tell the US Food and Drug Administration (FDA) about manipulated data? Why wait two months between documenting an initial issue and opening a nonconformance report (NCR)? And how harshly will FDA act? These questions and more are piling up for Novartis, following last week’s announcement that FDA is investigating the company for manipulating data linked to its $2.1 million gene therapy Zolgensma (onasemnogene abeparvovec-xioi). In Europe, regu...
  • Regulatory NewsRegulatory News

    Novartis’ Zolgensma Joins Growing List of Medicines to Lose Accelerated Assessment Status in EU

    The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recently announced its decision to remove Novartis’ spinal muscular atrophy gene therapy onasemnogene abeparvovec from its accelerated assessment program. CHMP did not announce its reasoning behind the decision, which effectively means that the treatment, approved in the US as Zolgensma and launched at a price of more than $2 million, will be reviewed in the EU in 210 days rather...
  • Regulatory NewsRegulatory News

    FDA’s No. 2 on RWE, Hype and Speeding Drug Development

    Just before PhRMA’s Innovation Day came to a close on Thursday, Novartis CEO Vas Narasimhan sat down with the US Food and Drug Administration’s (FDA) Principal Deputy Commissioner Amy Abernethy to discuss the rise of real-world evidence (RWE), what the technology and biopharma industries are over-hyping and why it’s so difficult to speed up drug development. Abernethy came to FDA less than a year ago from Roche’s Flatiron Health, which is at the forefront of the real-wo...
  • Regulatory NewsRegulatory News

    Regulating Digital Health: Novartis CEO Seeks Further Modernization

    Novartis CEO Vas Narasimhan told attendees of PhRMA’s Innovation Day on Thursday that as biopharmaceutical companies embrace digital technologies to speed the discovery and development of new medicines, regulators will have to adapt more quickly. “FDA has been, more than any other regulator in the world, interested in working with these technologies,” Narasimhan said. But he also opined that when the “rubber hits the road” and companies begin filing applications with th...
  • Regulatory NewsRegulatory News

    Gilead and Novartis Seek to Expand What FDA Considers as Real-World Data and Real-World Evidence

    Just over two months ago, the US Food and Drug Administration (FDA) released draft guidance explaining to drugmakers what constitutes real-world data (RWD) and real-world evidence (RWE) and how to submit such data to the agency. The draft guidance spells out what the agency considers to be an RWD source (i.e. electronic health record data, medical claims or billing data, etc.) and what relevant submissions may include RWE (i.e. single arm trials that use RWE as an ext...
  • Regulatory NewsRegulatory News

    FDA Approves First NDA Under Real-Time Oncology Review Pilot

    As the US grapples with whether Novartis’ new gene therapy for spinal muscular atrophy should cost $2.1 million, the US Food and Drug Administration (FDA) on Friday also quietly signaled that another Novartis drug would effectively kick off a new way for cancer drugs to be approved more quickly. The approval for the breast cancer treatment Piqray (alpelisib) in combination with fulvestrant and alongside a companion diagnostic — came more than three months ahead of its P...