• 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...
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    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...
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    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...
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    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    Just over two months ago, the US Food and Drug Administration (FDA) released draft guidance explaining to drugmakers what constitutes real-world data (RWD) and real-world evidence (RWE) and how to submit such data to the agency. The draft guidance spells out what the agency considers to be an RWD source (i.e. electronic health record data, medical claims or billing data, etc.)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...
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    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...
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    Novartis Sues Janssen Over False Advertisements for Psoriasis Drug

    Novartis filed a lawsuit on Friday in DC federal court claiming that Janssen has presented data on its severe plaque psoriasis drug in a false and misleading way in at least two presentations at conferences over the past several months. Novartis, which sold $2.8 billion worth of its plaque psoriasis treatment Cosentyx (secukinumab) in 2018, directly competes with Janssen, which sold $544 million worth of Tremfya (guselkumab) in 2018. In Janssen’s presentations, Novar...
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    Tropical Disease PRV Fix Didn’t Stop Novartis From Winning Another

    Back in 2017, a new law was enacted, known as the FDA Reauthorization Act (FDARA), which contained not only the new user fee programs for pharmaceuticals, medical devices, generics and biosimilars, but also a slight tweak to the tropical disease priority review voucher (PRV) program. The change was meant to ensure that companies winning PRVs (which means that they can sell such PRVs for potentially $100 million) actually performed some of the clinical work to bring ...
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    EU Regulatory Roundup: Rising Complexity Drives Sharp Fall in MHRA Pharmacovigilance Inspections

    Welcome to our European   Regulatory Roundup, our weekly overview of the top EU regulatory news.   EMA Advises Novartis on use of eSource Direct Data Capture in Clinical Trials   The European Medicines Agency (EMA) has outlined its views on eSource direct capture of clinical trial data in response to questions from Novartis. EMA sees no theoretical obstacles to the use of the technology in a way that complies with good clinical practices (GCPs), but thinks sponso...
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    Biosimilars Forum Calls for FDA Guidance to Address Misinformation

    Echoing a Pfizer petition and Novartis comments, the Biosimilars Forum said late last week that guidance from the US Food and Drug Administration (FDA) is needed to ensure reference product sponsors and other organizations communicate properly about biosimilars.   Amgen, which withdrew from the Forum recently, Roche’s Genentech and others have been singled out for misinformation campaigns that not only question the legal framework governing biosimilars but also the saf...
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    Asia Regulatory Roundup: Australia, Singapore Expand Generic Drug Work-Sharing Trial (12 December 2017)

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia. Australia, Singapore Expand Generic Drug Work-Sharing Trial A regulatory consortium featuring Australia and Singapore has expanded its generic medicine work-sharing program. The initiative allows companies to file for approval with multiple agencies, which then perform a coordinated assessment of the application before reaching individual decisions. Agencies in Aust...
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    House Reps Seek More Transparency on Novartis-CMS Pricing Deal for Newly Approved CAR-T Therapy

    Rep. Lloyd Doggett (D-TX) and other House Democrats on Wednesday sent a letter to Seema Verma, administrator of the Centers for Medicare & Medicaid Services (CMS), seeking to find out more information about an outcomes-based payment approach for Novartis’ newly approved chimeric antigen receptor T-cell (CAR-T) treatment Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). The outcomes-based pay...
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    FDA Panel Votes Unanimously in Favor of First CAR-T Cancer Therapy

    The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee on Wednesday voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of a new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy. Although the outside panel of experts raised questions about concerns with the safety and manufacturing of the Novartis treatment, known as CTL019 (tisagenlecleucel-T), the panel did not question the effi...