New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products

Innovation drives progress. When it comes to innovation in the development of new drugs and therapeutic biological products, FDA’s Center for Drug Evaluation and Research (CDER) supports the pharmaceutical industry at every step of the process. With its understanding of the science used to create new products, testing and manufacturing procedures, and the diseases and conditions that new products are designed to treat, FDA provides scientific and regulatory advice needed to bring new therapies to market.

The availability of new drugs and biological products often means new treatment options for patients and advances in health care for the American public. For this reason, CDER supports innovation and plays a key role in helping to advance new drug development. 

Each year, CDER approves a wide range of new drugs and biological products. Some of these products are innovative new products that never before have been used in clinical practice. Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace.

Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients. Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that have previously been approved by FDA. For example, CDER classifies biological products submitted in an application under section 351(a) of the Public Health Service Act as NMEs for purposes of FDA review, regardless of whether the Agency previously has approved a related active moiety in a different product. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act.

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New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products in 2014

FDA approves extended-release, single-entity hydrocodone product with abuse-deterrent properties

The U.S. Food and Drug Administration today approved Hysingla ER (hydrocodone bitartrate), an extended-release (ER) opioid analgesic to treat pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. Hysingla ER has approved labeling describing the product’s abuse-deterrent properties consistentwith the FDA’s 2013 draft guidance for industry, Abuse-Deterrent Opioids – Evaluation and Labeling.

Hysingla ER has properties that are expected to reduce, but not totally prevent, abuse of the drug when chewed and then taken orally, or crushed and snorted or injected. The tablet is difficult to crush, break or dissolve. It also forms a viscous hydrogel (thick gel) and cannot be easily prepared for injection. The FDA has determined that the physical and chemical properties of Hysingla ER are expected to make abuse by these routes difficult. However, abuse of Hysingla ER by these routes is still possible. It is important to note that taking too much Hysingla ER, whether by intentional abuse or by accident, can cause an overdose that may result in death.

Hysingla ER is not approved for, and should not be used for, as-needed pain relief. Given Hysingla ER’s risks for abuse, misuse and addiction, it should only be prescribed to people for whom alternative treatment options are ineffective, not tolerated or would be otherwise inadequate to provide sufficient pain management. As a single-entity opioid, Hysingla ER does not carry the serious liver toxicity risks associated with hydrocodone combination products containing acetaminophen. The FDA encourages health care professionals to review and consider all available information as part of their decision-making when prescribing opioid analgesics.

Strengths of Hysingla ER contain 20, 30, 40, 60, 80, 100 and 120 milligrams (mg) of hydrocodone to be taken every 24 hours. Doses of 80 mg per day and higher should not be prescribed to people who have not previously taken an opioid medication (opioid non-tolerant). While Hysingla ER contains larger amounts of hydrocodone compared to immediate-release hydrocodone combination products, the range of tablet strengths of Hysingla ER is comparable to existing approved ER opioids. 

For more information on the Approval CLICK HERE

USFDA : Manual of Policies and Procedures - Chemistry Review of Question-based-Review (QbR) of Submissions...

USFDA has updated Manual of Policies and Procedures Document Titled "Chemistry Review of Question-based-Review (QbR) of Submissions".

To Download the Document Please CLICK HERE

FDA approves first drug-coated angioplasty balloon catheter to treat vascular disease...

The U.S. Food and Drug Administration today approved Lumason (sulfur hexafluoride lipid microsphere) for patients whose ultrasound image of the heart (echocardiograms) are hard to see with ultrasound waves.

Lumason is a contrast agent made up of gas-filled microbubbles (or microspheres) that reflect the sound waves to enhance the image. Lumason helps a physician see a patient’s heart more clearly, allowing for clearer imaging of the left ventricle chamber and the smooth edge on the inside of the chambers of the heart, known as the endocardium. 

“Sometimes echocardiograms in certain patients are difficult for physicians to see and interpret,” said Libero Marzella, M.D., Ph.D., director of the Division of Medical Imaging Products in the FDA’s Center for Drug Evaluation and Research. “Today’s approval provides doctors with another option when performing contrast enhanced ultrasound.”

Lumason’s safety and efficacy were established in three clinical trials involving 191 patients with suspected cardiac disease whose echocardiograms were difficult to read and interpret. In all three studies, Lumason helped doctors see the lining of the left ventricle more clearly, with visual improvement observed in the majority of the patients who received a 2 milliliter dose of the agent. Lumason also helped independent reviewers in all three of the studies more clearly and completely see inside the patient’s left ventricle.

All microbubble contrast agents, including Lumason, carry a boxed warning about the risk of serious cardiopulmonary reactions, including fatal cardiac or respiratory arrest. The risk of these reactions may be increased among patients with certain heart conditions; most serious reactions occur within 30 minutes administration.

The most commonly reported side effects associated with Lumason were headache and nausea.

Lumason is marketed by Bracco Diagnostics Inc., located in Monroe Township, NJ.

EMEA : 15 Medicines set for EMEA Approval...

The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended a stream of new products for approval within the region, with a group of 15 now having jumped the final hurdle before an ultimate decision from regulators.

First up, Gilead’s Harvoni

(sofosbuvir/ledipasvir) has been backed for clearance to treat adults with hepatitis C (HCV). The drug belongs to a new generation of antiviral products for chronic HCV infection that have high cure rates, and marks the third new therapy for the disease put forward by the CMHP in the last few months.

Next, Laboratoire HRA Pharma’s Ketoconazole HRA (ketoconazole) has been recommended as a ‘new’ treatment for patients with the rare hormonal disorder Cushing’s syndrome. The drug, normally used to fight fungal infections (though no longer by mouth because of liver injury risk), has actually been used off-label to treat Cushing’s for more than 30 years, but it has never been formally approved for this indication.

Two cancer drugs have also won CHMP backing - Boehringer Ingelheim’s Vargatef (nintedanib) for the treatment of non-small cell lung cancer and Eli Lilly’s Cyramza (ramucirumab) for gastric cancer. The diagnostic agent Lymphoseek (tilmanocept) was also endorsed for the delineation and localisation of sentinel lymph nodes.

According to BI, Vargatef, when added to docetaxel, is the first lung cancer treatment to have provided over one-year overall survival for patients with advanced adenocarcinoma, after first-line chemotherapy. And with regard to Lilly’s biologic Cyramza, clinical data show that adding the drug to paclitaxel significantly boosted median overall survival in patients with advanced gastric cancer or gastroesophageal junction adenocarcinoma.

Elsewhere, the CHMP also recommended: Teva’s Egranli (balugrastim) for chemotherapy-induced neutropenia, as well as Almirall's Brimica Genuair and Duaklir Genuair (aclidinium/formoterol) for maintenance bronchodilator treatments to relieve symptoms of chronic obstructive pulmonary disease and asthma; Janssen’s Rezolsta (darunavir/cobicistat) for HIV; Eli Lilly’s Trulicity (dulaglutide) for type II diabetes; AstraZeneca’s Moventig(naloxegol) for opioid-induced constipation; and Generics (UK)’s generic Tadalafil Mylan for erectile dysfunction in adult males; 

Three hybrid applications have also received the thumbs up: Budesonide/Formoterol Teva and Vylaer Spiromax for the treatment of asthma and severe COPD, and Budesonide/Formoterol Teva Pharma B.V. for the treatment of asthma. Hybrid applications rely in part on the results of preclinical tests and clinical trials for a reference product and in part on new data, the Committee said.

USFDA approves AbbVie's HUMIRA® (adalimumab) for the Treatment of Pediatric Patients with Moderately to Severely Active Crohn's Disease

AbbVie announced today that the U.S. Food and Drug Administration (FDA) has approved HUMIRA^® (adalimumab) for reducing signs and symptoms, and achieving and maintaining clinical remission, in pediatric Crohn's disease patients 6 years of age and older when certain other treatments have not worked well enough. This FDA approval represents the eighth indication for HUMIRA in the United States and makes it the first and only biologic treatment approved for use in this patient population that can be administered at home.

-- HUMIRA is now indicated to treat pediatric patients 6 years of age and older with moderately to severely active Crohn's disease who have had an inadequate response to corticosteroids or immunomodulators such as azathioprine, 6-mercaptopurine, or methotrexate

-- HUMIRA is the first biologic approved in the U.S. that can be administered at home for the treatment of moderately to severely active Crohn's disease in pediatric patients
-- FDA granted HUMIRA Orphan Drug Designation for pediatric Crohn's disease

"Moderate to severe Crohn's disease is a serious chronic disorder that can impact children and adolescents in significant ways during this important stage of their lives," said Jeffrey S. Hyams, M.D., head, Division of Digestive Diseases, Hepatology and Nutrition, Connecticut Children's Medical Center. "The approval of HUMIRA for this patient population offers a new important treatment option for physicians and their patients that can help address this sometimes debilitating disease."

For information CLICK HERE

USFDA CDER Approves Eli-lilly's Trulicity (dulaglutide), a once-weekly subcutaneous injection for Type 2 Diabetes

Trulicity (dulaglutide), a once-weekly subcutaneous injection to improve glycemic control (blood sugar levels), along with diet and exercise, in adults with type 2 diabetes.

Type 2 diabetes affects about 26 million people and accounts for more than 90 percent of diabetes cases diagnosed in the United States. Over time, high blood sugar levels can increase the risk for serious complications, including heart disease, blindness, and nerve and kidney damage.

Trulicity is a glucagon-like peptide-1 (GLP-1) receptor agonist, a hormone that helps normalize blood sugar levels. The drug’s safety and effectiveness were evaluated in six clinical trials in which 3,342 patients with type 2 diabetes received Trulicity. Patients receiving Trulicity had an improvement in their blood sugar control as observed with reductions in HbA1c level (hemoglobin A1c is a measure of blood sugar control). 

Trulicity has been studied as a stand-alone therapy and in combination with other type 2 diabetes therapies, including metformin, sulfonylurea, thiazolidinedione, and prandial insulin. Trulicity should not be used to treat people with type 1 diabetes; those who have increased ketones in their blood or urine (diabetic ketoacidosis); those with severe stomach or intestinal problems; or as first-line therapy for patients who cannot be managed with diet and exercise.

Trulicity has a boxed warning that tumors of the thyroid gland (thyroid C-cell tumors) have been observed in rodent studies with Trulicity but that it is unknown whether Trulicity causes thyroid C-cell tumors, including a type of thyroid cancer called medullary thyroid carcinoma (MTC), in humans. Trulicity should not be used in patients with a personal or family history of MTC or in patients with multiple endocrine neoplasia syndrome type 2 (a disease in which patients have tumors in more than one gland in their body, which predisposes them to MTC). 

Trulicity is manufactured by Indianapolis-based Eli Lilly and Company.

For more information, please visit: Trulicity

USFDA granted accelerated approval to Keytruda (pembrolizumab) for treatment of advanced or unresectable melanoma..

The U.S. Food and Drug Administration today granted accelerated approval to Keytruda (pembrolizumab) for treatment of patients with advanced or unresectable melanoma who are no longer responding to other drugs.

Melanoma, which accounts for approximately 5 percent of all new cancers in the United States, occurs when cancer cells form in skin cells that make the pigment responsible for color in the skin. According to the National Cancer Institute, an estimated 76,100 Americans will be diagnosed with melanoma and 9,710 will die from the disease this year.

Keytruda is the first approved drug that blocks a cellular pathway known as PD-1, which restricts the body’s immune system from attacking melanoma cells. Keytruda is intended for use following treatment with ipilimumab, a type of immunotherapy. For melanoma patients whose tumors express a gene mutation called BRAF V600, Keytruda is intended for use after treatment with ipilimumab and a BRAF inhibitor, a therapy that blocks activity of BRAF gene mutations.

For further information, please visit: Keytruda.

USFDA Medical Device Approval of Low-Profile Visualized Intraluminal Support Device (LVIS and LVIS Jr.)

The FDA has approved the Low-Profile Visualized Intraluminal Support Device (LVIS and LVIS Jr.) to be marketed.  The LVIS Device is a stent and delivery system used to treat certain brain aneurysms. The stent is a self-expanding, nickel-titanium (nitinol) single wire braid. The delivery system consists of an introducer and delivery wire and is used to deliver the stent to the aneurysm. The stent keeps the soft platinum coils that are put into the aneurysm from slipping back into the main blood vessel.  

For more information, please see:http://www.fda.gov/MedicalDevices/ProductsandMedicalProcedures/DeviceApprovalsandClearances/Recently-ApprovedDevices/ucm411207.htm

USFDA approves new drug to treat a form of Gaucher disease...

August 19, 2014

The U.S. Food and Drug Administration today approved Cerdelga (eliglustat) for the long-term treatment of adult patients with the Type 1 form of Gaucher disease, a rare genetic disorder.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials to collect in the spleen, liver and bone marrow. The major signs of Gaucher disease include liver and spleen enlargement, low red blood cell counts (anemia), low blood platelet counts and bone problems.

Cerdelga is a hard gelatin capsule containing eliglustat that is taken orally. In patients with Gaucher disease Type 1, the drug slows down the production of the fatty materials by inhibiting the metabolic process that forms them. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States.

“Today’s approval offers another important treatment option for patients with Type 1 Gaucher disease,” said Amy G. Egan, M.D., M.P.H., deputy director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research. “In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency’s focus and commitment to the development of treatments for rare diseases.”

The safety and effectiveness of Cerdelga were evaluated in two clinical trials with 199 participants with Type 1 Gaucher disease.

In one randomized, double-blind, placebo-controlled, multicenter clinical trial the safety and effectiveness of Cerdelga were evaluated in 40 participants with Type 1 Gaucher’s disease who had not previously received enzyme replacement therapy. Subjects received the drug at a starting dose of 42 mg two times a day, with most receiving a dose of 84 mg two times a day after four weeks. Study participants continued the drug for nine months.

Compared to placebo, treatment with Cerdelga resulted in a greater reduction in spleen volume from baseline to the end of the study (by the 39th week), the trial’s primary endpoint. Cerdelga also resulted in greater improvement in liver volume, blood platelet count, and red blood cell (hemoglobin) level, compared to placebo.

The other trial sought to determine the safety and effectiveness of Cerdelga compared to enzyme replacement therapy in 159 participants with Type 1 Gaucher disease previously treated and stabilized on enzyme replacement therapy. Subjects in the trial received either the enzyme replacement therapy drug imiglucerase or Cerdelga. The trial demonstrated that treatment with Cerdelga resulted in similar stabilization of hemoglobin level, platelet count and spleen and liver volume as imiglucerase.

The most commonly observed side effects in the Cerdelga clinical trials were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

Cerdelga is manufactured by Cambridge, Massachusetts-based Genzyme.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Recent Medical Device "Cologuard" Approval by USFDA

The FDA has recently approved Cologuard to be marketed.  Cologuard is a laboratory test that measures DNA and blood in human stool that is not visibly apparent (occult hemoglobin). This test is for adults of either sex, 50 years or older, who are average-risk candidates for colorectal cancer screening.  

For more information, please see: http://www.fda.gov/MedicalDevices/ProductsandMedicalProcedures/DeviceApprovalsandClearances/Recently-ApprovedDevices/ucm410569.htm