FDA BREAKING NEWS

PRESS RELEASES FROM THE FDA

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CRIMINAL & CIVIL ACTIONS FILED AGAINST AEGERION PHARMACEUTICALS INC

Aegerion agrees to plead guilty & enter into a consent decree with FDA


SEPT 22 2017 -- WASHINGTON DC USA -- Today, Aegerion Pharmaceuticals Inc. agreed to plead guilty in the US District Court for the District of Massachusetts to two misdemeanor counts of violating the Federal Food, Drug, & Cosmetic Act (FD&C Act) involving the introduction of misbranded Juxtapid (lomitapide) into interstate commerce. 


In connection with this agreement, the criminal information filed today charged that Juxtapid was misbranded because Aegerion failed to comply with the requirements of the Juxtapid Risk Evaluation and Mitigation Strategy (REMS) program and because the drug’s labeling lacked adequate directions for all of Juxtapid’s intended uses. This agreement resolves a criminal investigation in which the U.S. FDA’s Office of Criminal Investigations played an important role.


Aegerion and one of its senior vice presidents, Charles M. Gerrits, PharmD, Ph.D also agreed to enter into a consent decree of permanent injunction with the US. The consent decree includes a comprehensive compliance program and legal tools for the FDA to ensure that the defendants comply with the law, subject to judicial oversight. The FDA’s Center for Drug Evaluation and Research was instrumental in crafting the compliance obligations agreed to in the consent decree.


“We sometimes require companies to put in place certain measures to more closely manage a drug’s risks when we don’t believe a medicine’s benefits would outweigh its side effects without these risk mitigation strategies. This might include requiring prescribers to undergo certain training on a drug’s risks, or having providers take steps to more closely monitor patients,” said FDA Commissioner Scott Gottlieb, M.D. 


“By failing to follow the safety requirements that Aegerion had agreed to, the company put patients’ lives at risk and didn’t honor the safety commitments they made as a condition of gaining approval for their drug. This is unacceptable. We will continue to pursue those who skirt the law, and flout patient safety and other post-market commitments, using all of the enforcement tools available to us. Post-market safety requirements are a key element of the FDA’s public health protections and we will ensure that they are fulfilled.”


As alleged in court documents filed by the U.S. Department of Justice today, rather than following the REMS requirement to distribute Juxtapid only for the narrow indication for which it was approved, Aegerion instead sought to render the diagnosis of homozygous familial hypercholesterolemia (HoFH), a rare disorder that that causes high cholesterol levels and early cardiovascular disease, as vague and indefinite as possible in order to extend the product use to additional patient populations. 


As part of the required REMS, Aegerion failed to give health care providers complete and accurate information about HoFH and how to properly diagnose it. Aegerion also filed a misleading REMS assessment report to the FDA in which the company failed to disclose that it was distributing Juxtapid using a definition of HoFH that was inconsistent with Aegerion’s pre-approval filings with the FDA and that did not correspond to any peer-reviewed clinical standard for diagnosing HoFH. 


As such, Aegerion failed to comply with the required elements under the REMS to assure safe use of Juxtapid, in violation of the FD&C Act. In addition, Aegerion management and sales personnel also distributed Juxtapid not only for the treatment of HoFH, but also as a treatment for high cholesterol generally, without adequate directions for such use.


Once entered by the court, the plea and consent decree will be part of a global resolution of multiple government investigations into Aegerion’s conduct with respect to the marketing and distribution of Juxtapid. This resolution was the result of a coordinated effort by the U.S. Department of Justice and several government agencies, including the FDA.


Juxtapid, approved in December 2012 as an adjunct therapy to treat HoFH, is subject to a REMS to ensure that its benefits outweigh its risks. The Juxtapid REMS requires Aegerion to, among other things, (1) educate prescribers about the risks of hepatotoxicity (liver toxicity) associated with the use of Juxtapid and the need to monitor patients who are treated with Juxtapid; and (2) ensure that Juxtapid is prescribed and dispensed only to those patients with a clinical or laboratory diagnosis consistent with HoFH.


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FDA CLEARS FIRST DUADENOSCOPE WITH DISPOSABLE DISTAL CAP


SEPT 20 2017 -- WASHINGTON DC USA -- The U.S. FDA today cleared the first duodenoscope with a disposable distal cap, a new feature that will improve access for cleaning and reprocessing. The Pentax ED34-i10T model duodenoscope is intended to provide visualization and access to the upper gastrointestinal (GI) tract to treat bile duct disorders and other upper GI problems.


“We believe the new disposable distal cap represents a major step towards lowering the risk of future infections associated with these devices,” said William Maisel, M.D., M.P.H., acting director of the Office of Device Evaluation in the FDA’s Center for Devices and Radiological Health. “Improving the safety of duodenoscopes is a top priority for the FDA, and we encourage companies to continue to pursue innovations that will help reduce the risk to patients.”


Duodenoscopes are used in more than 500,000 procedures each year as a less invasive way than traditional surgery to drain fluids from pancreatic and biliary ducts blocked by cancerous tumors, gallstones or other gastrointestinal conditions. The flexible lighted duodenoscope, which is threaded through the mouth into the top of the small intestine, is a complex medical device with many small working parts that can be difficult to clean. If not thoroughly cleaned and disinfected, the device can trap contaminated tissue or fluid in its crevices, transmitting infections between patients.


It is critical that hospitals and health care facilities meticulously follow manufacturer’s reprocessing instructions for cleaning and disinfecting duodenoscopes. This lowers the risk of spreading infections between patients. While the risk of infection transmission cannot be completely eliminated, the benefits of these devices continue to outweigh the risks in appropriately selected patients. 


In January 2017, the FDA issued a Safety Communication alerting health care providers about a design issue with an earlier version of the Pentax duodenoscope, the ED-3490TK. The communication identified the potential for cracks and gaps to develop in the adhesive that seals the duodenoscope’s distal cap onto the scope. These cracks and gaps could allow fluids and tissue to leak into the duodenoscope.


The new features of the Pentax ED34-i10T include a single use detachable and disposable distal cap, simpler user interface, improved ergonomics, improved image quality, and a reduced length. The FDA granted clearance of the ED34-i10T to Pentax of America.


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FDA APPROVES NEW TREATMENT FOR ADULTS WITH RELAPSED FOLLICULAR LYMPHOMA


SEPT 14 2017 -- WASHINGTON DC USA -- The U.S. FDA today granted accelerated approval to Aliqopa (copanlisib) for the treatment of adults with relapsed follicular lymphoma who have received at least two prior treatments known as systemic therapies.


“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them.”


Follicular lymphoma is a slow-growing type of non-Hodgkin lymphoma, a cancer of the lymph system. The lymph system is part of the body’s immune system and is made up of lymph tissue, lymph nodes, the spleen, thymus, tonsils and bone marrow. The National Cancer Institute at the National Insititutes of Health estimates that approximately 72,240 people in the US will be diagnosed with some form of non-Hodgkin lymphoma this year; approximately 20,140 patients with non-Hodgkin lymphoma will die from the disease in 2017.


The FDA granted the approval of Aliqopa to Bayer Healthcare Pharmaceuticals, Inc. Aliqopa is a kinase inhibitor that works by blocking several enzymes that promote cell growth.


Aliqopa received an Accelerated Approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need using clinical trial data that is thought to predict a clinical benefit to patients. Further clinical trials are required to confirm Aliqopa’s clinical benefit and the sponsor is currently conducting these studies.


Aliqopa also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.


Today’s approval of Aliqopa was based on data from a single-arm trial that included 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. The trial measured how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). In the trial, 59% of patients had a complete or partial response for a median 12.2 months.


Common side effects of Aliqopa include high blood sugar levels (hyperglycemia), diarrhea, decreased general strength and energy, high blood pressure (hypertension), low levels of certain white blood cells (leukopenia, neutropenia), nausea, lower respiratory tract infections, and low levels of blood platelets (thrombocytopenia).


Serious side effects include infections, high blood sugar levels (hyperglycemia), high blood pressure (hypertension), inflammation of the lung tissue (non-infectious pneumonitis), low levels of certain white blood cells (neutropenia), and severe skin reactions. Women who are pregnant or breastfeeding should not take Aliqopa because it may cause harm to a developing fetus or newborn baby.


Aliqopa was granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.


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FDA APPROVES FIRST BIOSIMILAR FOR TREATING CANCER

Mvasi, a biosimilar to the cancer drug Avastin, is approved for certain colorectal, lung, brain, kidney & cervical cancers


SEPT 14 2017 -- WASHINGTON DC USA -- The U.S. FDA today approved Mvasi (bevacizumab-awwb) as a biosimilar to Avastin (bevacizumab) for the treatment of multiple types of cancer. Mvasi is the first biosimilar approved in the U.S. for the treatment of cancer.


The FDA granted approval of Mvasi to Amgen, Inc. Avastin was approved in February 2004 and is manufactured by Genentech, Inc.


“Bringing new biosimilars to patients, especially for diseases where the cost of existing treatments can be high, is an important way to help spur competition that can lower healthcare costs and increase access to important therapies,” said FDA Commissioner Scott Gottlieb, M.D. “We’ll continue to work hard to ensure that biosimilar medications are brought to the market quickly, through a process that makes certain that these new medicines meet the FDA’s rigorous gold standard for safety and effectiveness.”


Mvasi is approved for the treatment of adult patients with certain colorectal, lung, brain, kidney and cervical cancers. Specifically, the approved indications include:


Metastatic colorectal cancer, in combination with intravenous 5-fluorouracil-based chemotherapy for first- or second-line treatment. Mvasi is not indicated for the adjuvant treatment of surgically resected colorectal cancer.


Metastatic colorectal cancer, in combination with fluoropyrimidine-irinotecan- or fluoropyrmidine-oxaliplatin-based chemotherapy for the second-line treatment of patients who have progressed on a first-line bevacizumab product-containing regimen. Mvasi is not indicated for the adjuvant treatment of surgically resected colorectal cancer.


Non-squamous non-small cell lung cancer, in combination with carboplatin and paclitaxel for first line treatment of unresectable, locally advanced, recurrent or metastatic disease.


Glioblastoma with progressive disease following prior therapy, based on improvement in objective response rate. No data is available demonstrating improvement in disease-related symptoms or survival with bevacizumab products.


Metastatic renal cell carcinoma, in combination with interferon alfa.


Cervical cancer that is persistent, recurrent, or metastatic, in combination with paclitaxel and cisplatin or paclitaxel and topotecan.

Health care professionals should review the prescribing information in the labeling for detailed information about the approved uses.


Biological products are generally derived from a living organism and can come from many sources, such as humans, animals, microorganisms or yeast. A biosimilar is a biological product that is approved based on data showing that it is highly similar to an already-approved biological product and has no clinically meaningful differences in terms of safety, purity and potency (i.e., safety and effectiveness) from the reference product, in addition to meeting other criteria specified by law.


The FDA's approval of Mvasi is based on review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamics data, clinical immunogenicity data and other clinical safety and effectiveness data that demonstrates Mvasi is biosimilar to Avastin. It has been approved as a biosimilar, not as an interchangeable product.


Common expected side effects of Mvasi include nose bleeds (epistaxis), headache, high blood pressure (hypertension), inflammation of the nasal cavity (rhinitis), high levels of protein in the urine (proteinuria), taste alteration, dry skin, rectal bleeding (hemorrhage), excessive tear production (lacrimation disorder), back pain and skin irritation (exfoliative dermatitis).


Serious expected side effects of Mvasi include holes in or abnormal connection between two organs (perforation or fistula), blood clot formation (arterial and venous thromboembolic events), hypertension, problems in brain function or structure (posterior reversible encephalopathy syndrome), high levels of protein in the urine (proteinuria), infusion-related reactions and loss of function of the ovaries (ovarian failure). Patients should stop using Mvasi if these side effects become severe or life-threatening. Women who are pregnant should not take Mvasi because it may cause harm to a developing fetus.


Like Avastin, the labeling for Mvasi contains a Boxed Warning to alert health care professionals and patients about an increased risk of holes in the stomach and intestines (gastrointestinal perforations); surgery and wound healing complications; and severe or fatal pulmonary, gastrointestinal, central nervous system and vaginal bleeding (hemorrhage). Patients should stop using Mvasi if gastrointestinal perforation occurs. Patients should not take Mvasi in the 28 days prior to and after elective surgery, and until the surgical wound is fully healed. Patients should stop using Mvasi if a surgical incision breaks open (wound dehiscence). Mvasi should not be given to patients with severe hemorrhage or in patients who cough up blood (hemoptysis).

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FDA APPROVES NEW ANTIBACTERIAL DRUG FOR TREATING UTI


AUG 29 2017 -- WASHINGTON DC USA -- The U.S. FDA today approved Vabomere for adults with complicated urinary tract infections (cUTI), including a type of kidney infection, pyelonephritis, caused by specific bacteria. Vabomere is a drug containing meropenem, an antibacterial, and vaborbactam, which inhibits certain types of resistance mechanisms used by bacteria.


The FDA granted approval of Vabomere to Rempex Pharmaceuticals.


“The FDA is committed to making new safe and effective antibacterial drugs available,” said Edward Cox, M.D., director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research. “This approval provides an additional treatment option for patients with cUTI, a type of serious bacterial infection.”


The safety and efficacy of Vabomere were evaluated in a clinical trial with 545 adults with cUTI, including those with pyelonephritis. At the end of intravenous treatment with Vabomere, approximately 98% of patients treated with Vabomere compared with approximately 94% of patients treated with piperacillin/tazobactam, another antibacterial drug, had cure/improvement in symptoms and a negative urine culture test. Approximately seven days after completing treatment, approximately 77% of patients treated with Vabomere compared with approximately 73% of patients treated with piperacillin/tazobactam had resolved symptoms and a negative urine culture. 


The most common adverse reactions in patients taking Vabomere were headache, infusion site reactions and diarrhea. Vabomere is associated with serious risks including allergic reactions and seizures. Vabomere should not be used in patients with a history of anaphylaxis, a type of severe allergic reaction to products in the class of drugs called beta-lactams.


To reduce the development of drug-resistant bacteria and maintain the effectiveness of antibacterial drugs, Vabomere should be used only to treat or prevent infections that are proven or strongly suspected to be caused by susceptible bacteria. 


Vabomere was designated as a qualified infectious disease product (QIDP). This designation is given to antibacterial products that treat serious or life-threatening infections under the Generating Antibiotic Incentives Now (GAIN) title of the FDA Safety and Innovation Act. As part of its QIDP designation, Vabomere received a priority review.  


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FDA APPROVES TREATMENT FOR CHRONIC GRAFT VS. HOST DISEASE


AUG 02 2017 -- WASHINGTON DC USA -- The U.S. FDA today expanded the approval of Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after failure of one or more treatments. This is the first FDA-approved therapy for the treatment of cGVHD.


cGVHD is a life-threatening condition that can occur in patients after they receive a stem cell transplant from blood or bone marrow, called hematopoietic stem cell transplantation (HSCT), to treat certain blood or bone marrow cancers. cGVHD occurs when cells from the stem cell transplant attack healthy cells in a patient’s tissues. Symptoms of cGVHD can occur in the skin, eyes, mouth, gut, liver and lungs. The condition is estimated to occur in 30-70% of all patients who receive HSCT.


“Patients with cGVHD who do not respond to other forms of therapy—typically corticosteroids to suppress their immune system—now have a treatment option specifically indicated to treat their condition,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval highlights how a known treatment for cancer is finding a new use in treating a serious and life-threatening condition that may occur in patients with blood cancer who receive a stem cell transplant.”


The efficacy and safety of Imbruvica for the treatment of cGVHD were studied in a single-arm trial of 42 patients with cGVHD whose symptoms persisted despite standard treatment with corticosteroids. Most patients’ symptoms included mouth ulcers and skin rashes, and more than 50% of patients had two or more organs affected by cGVHD. In the trial, 67% of patients experienced improvements in their cGVHD symptoms. In 48% of patients in the trial, the improvement of symptoms lasted for up to five months or longer.


Common side effects of Imbruvica in patients with cGVHD include fatigue, bruising, diarrhea, low levels of blood platelets (thrombocytopenia), muscle spasms, swelling and sores in the mouth (stomatitis), nausea, severe bleeding (hemorrhage), low levels of red blood cells (anemia) and lung infection (pneumonia).


Serious side effects of Imbruvica include severe bleeding (hemorrhage), infections, low levels of blood cells (cytopenias), irregular heartbeat (atrial fibrillation), high blood pressure (hypertension), new cancers (second primary malignancies) and metabolic abnormalities (tumor lysis syndrome). Women who are pregnant or breastfeeding should not take Imbruvica because it may cause harm to a developing fetus or a newborn baby.

Imbruvica, a kinase inhibitor, was previously approved for certain indications in treating chronic lymphocytic leukemia, Waldenström’s macroglobulinemia and marginal zone lymphoma, as well as under accelerated approval status for mantle cell lymphoma.


The FDA granted this application Priority Review and Breakthrough Therapy designations. Imbruvica also received Orphan Drug designation for this indication, which provides incentives to assist and encourage the development of drugs for rare diseases.


The FDA granted the approval of Imbruvica to Pharmacyclics LLC.


PHOTO DETAILS:


The adjacent 2008 photograph (took left corner) shows a CDC scientist as he was examining a culture flask containing Madin-Darby Canine Kidney epithelial cells, and examining it for any signs of growth in a stock of Influenza virus.


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