• Regulatory NewsRegulatory News

    'Historic Action': FDA Approves First CAR-T Therapy

    The US Food and Drug Administration (FDA) on Wednesday approved the first gene therapy, Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Dubbed an "historic action" by the agency, the approval comes a little more than a month after FDA's Oncologic Drugs Advisory Committee voted unanimously , 10 to 0, in favor of the benefit-risk profile for the first of this new kind of cancer th...
  • Regulatory NewsRegulatory News

    Bill Wants Drugs Approved in Europe to be Available More Quickly to US Patients

    New legislation introduced in the US House of Representatives this week would require the US Food and Drug Administration (FDA) to expedite the review and approval of pharmaceutical products already approved in Europe. Background The bill, the Speeding Access to Already Approved Pharmaceutical Act , is sponsored by Reps. Tim Ryan (D-OH) and Steve Stivers (R-OH), who introduced the same bill in June 2014 . As with the original, the bill targets something known in th...
  • Regulatory NewsRegulatory News

    How Many Drug Companies Manage to Get Two New Drugs Approved by FDA? Not Many

    The development of a new pharmaceutical or biopharmaceutical product is a notoriously difficult process. According to industry data compiled by the Tufts Center for the Study of Drug Development, only about one in 10 drugs entering into Phase I clinical trials will ever be approved by the US Food and Drug Administration (FDA)—and those are just the companies whose drugs actually made it to the clinical development stage.  Getting a drug approved, in other words, can feel ...
  • New Brain Hemorrhage Treatment Intended as Improvement Over Existing Risky One

    Regulators with the US Food and Drug Administration (FDA) announced on Tuesday the approval of Arbor Pharmaceuticals' drug Nymalize (nimodipine), a drug intended to treat brain hemorrhaging in patients. FDA's 14 May 2013 statement explained that the agency has received reports over the years regarding "serious and sometimes fatal consequences" associated with using intravenous (IV) injections of nimodipine capsules, including death, cardiac arrest and severe decreases in...
  • Ultra-Rare Disease gets New FDA-Approved Therapy Intended to Make Treatment more Tolerable

    US regulators have approved a new product intended to treat an extremely rare disease known as nephropathic cystinosis, a disease that affects fewer than 3,000 patients worldwide and causes a buildup of cysteine in the body that can be fatal if not treated early in life. The drug, Procysbi, treats the condition by providing a delayed-release formulation that delivers cysteamine to patients in a new method. The drug's sponsor, Raptor Pharmaceuticals, explains on its websi...
  • FDA Clears Mayo Clinic to Manufacture PET Drug

    Regulators with the US Food and Drug Administration (FDA) have approved the manufacture of an existing positron emission tomography (PET) drug for use at a new facility, the agency has announced. The drug, Choline C 11 Injection, is used to help detect the presence of recurrent prostate cancer. The drug is administered to patients intravenously, and is specifically indicated for patients whose blood prostate specific antigen (PSA) levels have risen despite earlier trea...
  • Astellas' Prostate Cancer Drug Receives Priority FDA Approval

    A new drug manufactured by California-based Astellas Pharma promises to help treat aggressive, late-stage prostate cancer that has evaded other treatments, the US Food and Drug Administration (FDA) said in a statement approving the drug on 31 August. The drug, Xtandi (enzalutamide), was approved under FDA's priority review program, and was reviewed in just three months-half the time allotted to FDA under the priority review program. Xtandi was subject to a clinical tri...
  • Blockbuster Asthma Drug Singulair Receives Generic Competition

    Generic versions of Merck's pharmaceutical blockbuster Singulair (montelukast sodium) are now available to consumers, making it the latest product to fall off the so-called 'patent cliff,' the US Food and Drug Administration (FDA) announced on 3 August . The drug is used by asthma and allergy sufferers to reduce inflammation, and was Merck's most lucrative, bringing in sales of up to $5 billion per year . The company is expected to lose much of its market share to its ...
  • UK: New Anti-clotting Drug Cleared for NHS Use

    According to final guidance issued 25 July by the National Institute of Health and Clinical Excellence (NICE) -- the UK's health technology assessment entity -- Bayer's Xarelto (rivaroxaban) has been cleared for use by the National Health Service (NHS) for treatment of deep vein thrombosis (DVT).   NICE officials said a key factor in the positive assessment was that the new oral anticoagulant is easier to use than warfarin because it does not require the...
  • Feature ArticlesFeature Articles

    Approval of Jakafi (ruxolitinib) Based on a Home-Grown, Patient-Reported Outcome Instrument: A Case Study

    Jakafi® (ruxolitinib) was approved 1 by the US Food and Drug Administration (FDA) on 16 November 2011 for the treatment of intermediate or high-risk myelofibrosis (MF) including primary MF, post-polycythemia vera MF (PPV-MF) and post-essential thrombocythemia MF (PET-MF). Prior to this time, there were no approved products, and therefore no precedent for regulatory endpoints in this orphan disease. MF is a highly symptomatic 2 myeloproliferative neoplasm (MPN), cha...
  • US Regulators Approve First Obesity Drug in 13 Years

    In a possible shift its existing policy framework, the US Food and Drug Administration (FDA) has approved Arena Pharmaceuticals' anti-obesity drug Belviq (lorcaserin), making it the first anti-obesity drug to be approved in 13 years. The drug was subject to concerns-as are many anti-obesity drugs-about cardiovascular risks to patients, and at least one public advocacy group had urged FDA not to approve the drug based on those concerns. FDA seemed to take those risks...
  • Senate Passes FDA User Fee Legislation, Bill Now Moves to White House

    In an expected development, the US Senate on Tuesday (26 June) voted overwhelmingly to approve the Food and Drug Administration Safety and Innovation Act (FDASIA) , advancing the bill to the White House where it is expected to receive approval from President Barack Obama in July. The 92-4 vote followed months of negotiating between the House of Representatives, the Senate, the US Food and Drug Administration (FDA), the pharmaceutical industry, the medical device i...