Pilot Program Between FDA, EMA Includes Parallel QbD Reviews

Under a new pilot program, NDAs submitted to the FDA and European Medicines Agency (EMA) with Quality by Design (QbD) components will undergo parallel review by the agencies. The pilot, which will begin April 1, will allow parallel evaluation of relevant development and manufacturing data components, known as QbD, the FDA said Wednesday. The pilot will run until March 31, 2014, at which time the agencies will assess the program and issue a joint report, the EMA says. The dual review will not expedite the approval process, as reviews will take the standard time, FDA spokeswoman Morgan Liscinsky told WDL.

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Novartis Forced to Wait Three Additional Months for Indacaterol Decision

The FDA is asking for additional time to review data on Novartis’ once-daily bronchodilator indacaterol, adding another twist to what’s being called a potential blockbuster drug. However, Novartis said Wednesday the FDA moved a final decision date on indacaterol from April to July. The FDA did not request additional data, the company said. An FDA advisory committee recommended approval of a 75-mcg dose — rejecting a 150-mcg version — finding little benefit in the higher strength for treatment of chronic obstructive pulmonary disease.

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FDA Posts Track-and-Trace Workshop Information

 FDA has posted the slides from the Track-and-Trace Workshop held in February (which can be found in the “presentations” section at the bottom of the webpage). Additional details pertaining to the workshop, including the Workshop agenda and the announcement, can be found on this website.

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In a Shocking Move Proposed Budget for 2012 Cuts Biosimilar Exclusivity

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Biosimilars: Critical FDA Meeting Setting a Plan of Action for Approval

The FDA regulates biologic drugs under the 1944 Public Health Service Act. Congress gave the agency the power to approve generic versions of traditional chemical drugs in 1984. The streamlined process will expand the market for biologics, though it is not expected to cut drug costs as much as traditional generics, the Congressional Budget Office in a 2008 report.

The FDA is meeting this week Silver Spring, Maryland to weigh factors for approving the new products. Name-brand drug makers argued that biosimilar manufacturers should be forced to use the streamlined process because it requires turning over more data.

Companies are being encouraged to look at a more steam lined process that can cut the time it takes to do clinical trials, helping companies that want the FDA to use one set of data to see how a drug works on different conditions.

It is anticipated that the FDA will allow generic-drug makers to use either the new system, which requires them to give proprietary data to brand-name drug makers at the point they seek approval, or an older system used to clear novel biologics, which keeps that information private.   While the FDA wasn’t expected to take an immediate stand on the process, the new law accommodates both.

While the process written into the health law, signed in March it was designed to speed up approvals by potentially allowing shorter and smaller clinical trials however, it may also open generic companies to expensive and time-consuming patent litigation.

D2 will continue to stay tuned to this exciting new marketplace.

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BLA vs NDA What are the differences?

We are seeing more and more specialty products coming to market via the Biologic License Application ( BLA) route, so what are the key differences?  For a layman's definition look below:

"After clinical trials have shown that the new agent is safe and effective, there is reason to make the agent generally available to patients and physicians. The formal process in the U.S. by which this occurs is the approval by FDA of a marketing application (New Drug Application for cytotoxic/cytostatic agents or a Biologic License Application for biological agents) submitted by a private firm.  The applicant seeks approval from FDA for one or more specific indication(s). Review and approval of an NDA or BLA are based on the demonstration of safety and efficacy assessed from detailed reports of the clinical trials; particularly randomized controlled studies. The contribution of a new agent in the treatment of a disease is demonstrated unambiguously if the agent is the only variable between the treatments."

I'll add something below from fda.gov

"In june 30, 2003 FDA transferred some of the therapeutic biological products that had been reviewed and regulated by the Center for Biologics Evaluation and Research (CBER) to the Center for Drug Evaluation and Research (CDER).

Categories of Biological Products Transferred to CDER :

>Monoclonal antibodies for in vivo use.
>Proteins intended for therapeutic use, including cytokines (e.g. interferons), enzymes (e.g. thrombolytics), and other novel proteins, except for those that are specifically assigned to CBER (e.g., vaccines and blood products). This category includes therapeutic proteins derived from plants, animals, or microorganisms, and recombinant versions of these products.
>Immunomodulators: proteins or peptides that are not antigen specific (e.g., cytokines, growth factors, chemokines, etc.) that are intended to treat disease by inhibiting or modifying a pre-exisiting immune response; and proteins or peptides intended to act in antigen-specific fashion to treat or prevent autoimmune diseases by inhibiting or modifying pre-existing immune responses.
>Growth factors, cytokines, and monoclonal antibodies intended to mobilize, stimulate, decrease or otherwise alter the production of cells in vivo.1 This category includes growth factors, cytokines, and monoclonal antibodies, as well as non-biological agents, administered as mobilizing agents for their direct therapeutic effect on the recipient, as well as growth factors, cytokines, and monoclonal antibodies administered for the purpose of subsequently harvesting the mobilized, stimulated, decreased or otherwise altered cells for use in a human cellular or tissue-based product (HCT/P).

Categories of Biological Products Remaining in CBER

>Cellular products, including products composed of human, bacterial or animal cells (such as pancreatic islet cells for transplantation), or from physical parts of those cells (such as whole cells, cell fragments, or other components intended for use as preventative or therapeutic vaccines).
>Gene therapy products. Human gene therapy/gene transfer is the administration of nucleic acids, viruses, or genetically engineered microorganisms that mediate their effect by transcription and/or translation of the transferred genetic material, and/or by integrating into the host genome. Cells may be modified in these ways ex vivo for subsequent administration to the recipient, or altered in vivo by gene therapy products administered directly to the recipient.
>Vaccines and vaccine-associated products: products, regardless of their composition or method of manufacture, intended to induce or enhance a specific immune response to prevent or treat a disease or condition, or to enhance the activity of other therapeutic interventions.
>Allergenic extracts used for the diagnosis and treatment of allergic diseases and allergen patch tests.
>Antitoxins, antivenins, and venoms
>Blood, blood components, plasma derived products (for example, albumin, immunoglobulins, clotting factors, fibrin sealants, proteinase inhibitors), including recombinant and transgenic versions of plasma derivatives, (for example clotting factors), blood substitutes, plasma volume expanders, human or animal polyclonal antibody preparations including radiolabeled or conjugated forms, and certain fibrinolytics such as plasma-derived plasmin, and red cell reagents.
>Human cells, tissues and cellular and tissue-based products (HCT/P’s). This category includes HCT/P’s containing cells that have been harvested following in vivo administration of a CDER-regulated growth factor, cytokine, or monoclonal antibody,2 as well as HCT/P’s requiring ex vivo manipulation.

For more information go to:  http://www.fda.gov/Drugs/informationondrugs/ucm079436.htm

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FDA Revised Bar Code Guidance

 FDA announced the availability of a draft document, “Guidance for Industry: Bar Code Label Requirements—Questions and Answers (Question 12 Update),” in the September 7 Federal Register. The previous Bar Code Guidance was issued on October 5, 2006 (71 FR 58739); the Agency is proposing to amend question 12 (Q12) to provide recommendations to manufacturers of licensed vaccines in connection with the use of alternative coding technologies.

FDA believes that an alternative regulatory program, made up of technology such as two-dimensional symbology, could render the use of linear bar codes unnecessary for patient safety and enhance healthcare providers' ability to comply with the National Childhood Vaccine Injury Act of 1986. FDA would consider granting a request for exemption to the bar code requirement. The guidance can be found on the FDA website.

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FDA Decision regarding Questcor Pharma

In less than a week, the Food and Drug Administration will take a final decision on Questcor Pharmaceuticals Inc.'s (QCOR) H.P. Acthar Gel for the proposed expanded indication  of infantile spasms.

 The regulatory agency was originally scheduled to rule on Acthar's expanded indication in June. But the deadline was extended in order to review information regarding labeling and potential post-approval commitments that they solicited from Questcor.

 Infantile spasm, or IS, is a seizure disorder of early childhood also known as West Syndrome. The onset is predominantly in the first year of life, typically between 3 to 6 months. The typical pattern of IS is a sudden bending forward and stiffening of the body, arms and legs, although there can also be arching of the torso.

 Sabril is the first and the only FDA-approved drug for infantile spasms. The drug developed by Danish pharmaceutical company H.Lundbeck A/S (HLUKY.PK) was approved last August and was launched in the U.S. last September with an extensive REMS (Risk Evaluation Mitigation Strategy) program as required by the FDA.

The proposed indication of Questcor's H.P. Acthar Gel for infantile spasms cleared the penultimate regulatory hurdle by winning the FDA panel backing this May. The regulatory agency's Advisory Committee for Peripheral and Central Nervous System Drugs voted 22 to 1 in favor of H.P. Acthar Gel's efficacy as a treatment for patients with IS, and voted 20 to 1 in favor of the drug's safety at an effective dosing regimen.

H.P. Acthar gel is an injectable drug that is already approved in the U.S. by the FDA for the treatment of certain disorders with an inflammatory component, including the treatment of exacerbations associated with multiple sclerosis and to induce a diuresis or a remission of proteinuria in the nephrotic syndrome without uremia of the idiopathic type or that is due to lupus erythamatosus. Pursuant to guidelines published by the American Academy of Neurology and the Child Neurology Society, many child neurologists use Acthar to treat infants afflicted with infantile spasms.

The FDA usually follows the recommendations of its panel though not mandatory. A final decision on whether or not to approve H.P. Acthar Gel for infantile spasm is slated to be taken by the FDA by September 11. Upon approval, the company plans to launch Acthar in the treatment of IS during the Child Neurology Society Annual Meeting in October.

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D2’s Own Joe Kasid Featured on Specialty Pharmacy News regarding Avastin

D2's own Joe Kasid a former exectutive with Genetech provided comments to Specialty Pharmacy News regarding Avastin.  His keen objective insights provide readers critical information regarding future developments on this specialtyproduct, key quotes below:

"But it’s good practice for health plans to understand the indication a claim is for when there are multiple uses of a therapy, Joe Kasid, director of specialty trade operations for D2 Pharma Consulting, LLC, adds. Plans, he says, want to make sure that providers get “the right drug to the right patient for the right indication because the most expensive drug is one that’s not effective or used incorrectly,” such as when a patient is not adherent to a regimen. If the FDA retracts the approval, health plans most certainly will face a coverage quandary, contends Kasid.  “The question out there is if the FDA revokes the use of Avastin in breast cancer, and patients currently on it are receiving benefit from it, does a health plan require the physician to stop that therapy?” asks Kasid, who worked for Genentech for 15 years. “This is an ethical as well as a critical question.”  If plans do decide to deny claims for Avastin, physicians can still appeal the decision by providing a statement of medical necessity, points out Kasid. “Avastin has been a great product in the armamentarium of oncologists,” he says. “It would appear that this [FDA] administration is taking a little different tack on how they look at the approval and effectiveness of products.  Is this good or bad? It remains to be seen what the fallout would be if it’s revoked.” Part of that fallout would undoubtedly be that “payers will scrutinize Avastin use even more,” Kasid maintains. And if the drug’s use for one indication has been questioned, that could lead to the  determination that Avastin is “maybe not the best thing since sliced bread for this indication either.” 

Specialty Rx Facts readers are encouraged to obtain your own copy of Specialty Pharmacy News.  

Joe Kasid can be reached at joe.kasid@d2rx.com



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FDA warns of brain problem with Glaxo seizure drug

Thu Aug 12, 2:55 pm ET


WASHINGTON – Federal health regulators are warning doctors and patients that an anti-seizure drug from GlaxoSmithKline PLC can cause rare inflammation of the brain and spinal cord.

The Food and Drug Administration said Thursday it is working with the British drugmaker Glaxo to add new warnings and labeling information to the company's drug Lamictal.

The agency said it has received reports of 40 cases of aseptic meningitis between 1994, when Lamictal was approved, and last November. Thirty-five patients needed to be hospitalized, the agency said in a statement. The symptoms usually emerged within the first month and a half of treatment.

Aseptic meningitis is a dangerous inflammation of the brain and spinal cord that can cause headache, fever, chills and vomiting. The problem can be caused by viruses, toxins and certain medications. Treatment for the illness, which usually resolves itself in two weeks, generally involves pain medications.

Lamictal is part of the anti-seizure family of medications. The drug, which posted sales of $778 million last year, is also approved by the FDA to treat manic depression.

GlaxoSmithKline said in a statement it will add language about the risk to a medication guide distributed to patients. According to the company, aseptic meningitis is a "very rarely reported event."

The company's U.S-traded shares rose 74 cents, or 2 percent, to $37.20 in afternoon trading.


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