U.S. may speed approval of "breakthrough" drugs

Under a proposal likely to become law later this year, U.S. drug regulators would be able to  label promising treatments as "breakthorugh" therapies to speed their path to approval. 

At Reuters.com, journalist Anna Yukhananov writes that the proposal would allow drug regulators to "work with companies to speed up clinical trials, for example by testing the drugs for a shorter time or enrolling fewer patients...The plan fits with President Barack Obama's aim to foster innovation as a means of spurring job creation, and may pacify some critics who say the FDA lags European countries in approving new medicines or medical devices."

Yukhananov quoted Dr. Janet Woodcock, head of the FDA's drugs center, who in March asked reporters, "What happens when you have a breakthrough drug that shows an effect that's never been seen before? If we'd done business as usual during the AIDS epidemic, we would have never controlled that epidemic."

Also quoted was Jeff Allen, executive director of Friends of Cancer Research, who commented, "The most promising drugs show an effect early. [But] there's a mentality mong drug developers or FDA reviewers that you have to go through this multi-step approach" to get a drug to market. We're hoping to encourage getting away from that."

Read more at Reuters.com. 

Listing of Upcoming Meetings from FDA Live - 4/30/2012

FDALive is the World's Leading Source for FDA Broadcasting. Below is the list of upcoming meetings, current as of April 30, 2012:

Pediatric AdComm

Meeting Date: 5/7/12-5/8/12  

Agenda: On May 7, 2012, the Pediatric Advisory Committee will meet to discuss pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act, for Differin Lotion (adapalene), Dulera Inhalation Aerosol (mometasone furoate and formot...

 

Arthritis AdComm

Meeting Date: 5/8/12-5/8/12  

The committee will discuss supplemental biologics license application 125249, ARCALYST (rilonacept) injection, Regeneron Pharmaceuticals, Inc., for the following proposed indication: "ARCALYST (rilonacept) is an interleukin-1 blocker indicated for the prevention of gout flares during initiation o...

 

Arthritis AdComm

Meeting Date: 5/9/12-5/9/12  

The committee will discuss new drug application (NDA) 203214, tofacitinib tablets, Pfizer Inc., for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs....

Antiviral Drugs AdComm

Meeting Date: 5/10/12-5/10/12  

The committee will discuss an efficacy supplement for new drug application (NDA) 21-572, TRUVADA (emtricitabine/tenofovir disoproxil fumarate) Tablet, submitted by Gilead Sciences, Inc. The supplemental application proposes an indication for Pre-Exposure Prophylaxis (PrEP) to reduce the risk...

Endocrinologic and Metabolic Drugs AdComm

Meeting Date: 5/10/12-5/10/12  

The committee will discuss the safety and efficacy of new drug application (NDA) 22–529 (lorcaserin hydrochloride) tablets, manufactured by Arena Pharmaceuticals, Inc., as an adjunct to diet and exercise for weight management in patients with a body mass index (BMI) equal to or greater than ...

 

Gastroenterology and Urology Devices Panel of the Medical Devices AdComm

Meeting Date: 5/10/12-5/11/12  

On May 10 and 11, 2012, the committee will discuss general issues related to medical devices intended for obese patients. The committee will provide recommendations regarding trial design for clinical studies to evaluate the safety and effectiveness of weight loss devices placed either endoscopic...

 

Antiviral Drugs AdComm

Meeting Date: 5/11/12-5/11/12  

The committee will discuss new drug application (NDA) 203-100, for a fixeddose combination tablet of elvitegravir/cobicistat/emtricitabine/tenofovir disoproxil fumarate, submitted by Gilead Sciences, Inc. The application proposes an indication for the treatment of HIV-1 infection in adults who ar...

Cardiovascular and Renal Drugs AdComm

Meeting Date: 5/23/12-5/23/12  

On May 23, 2012, the Committee will discuss supplemental new drug application (sNDA) 202439/S-002 XARELTO (rivaroxaban), submitted by Janssen Pharmaceuticals, Inc. to reduce the risk of thrombotic cardiovascular events in patients with acute coronary syndrome (ACS) [ST elevation myocardial infarctio...

 

Peripheral and Central Nervous System Drugs AdComm

Meeting Date: 5/24/12-5/24/12  

On May 24, 2012, the committee will discuss new drug application (NDA) 202737 for tafamidis meglumine capsules, proposed trade name VYNDAQEL, submitted by FoldRx Pharmaceuticals, Inc. a subsidiary of Pfizer, Inc. The proposed indication is for the treatment of transthyretin (TTR) familial amyloid po...

Circulatory System Devices Panel of the Medical Devices AdComm

Meeting Date: 5/24/12-5/24/12  

On May 24, 2012, the committee will discuss current knowledge about the safety and effectiveness of the AMPLATZER ASO Device & Gore HELEX ASD Occluder as transcatheter Atrial Septal Defect (ASD) occluders used for the closure of secundum atrial septal defects. The AMPLATZER Septal Occluder (ASO) ...

 

Assessment of Analgesic Treatment of Chronic Pain--A Public Workshop

Meeting Date: 5/30/12-5/31/12  

The purpose of the meeting is to provide a forum to discuss the available data on the use of analgesics in the treatment of CNCP, beginning with a discussion of the underlying mechanisms of chronic pain and the epidemiology of chronic pain in the United States. Next, data on the efficacy of opioi...

 

Gastrointestinal Drugs AdComm

Meeting Date: 5/31/12-5/31/12  

The committee will discuss new drug application (NDA) 200-436, synthetic human secretin, sponsored by Repligen Corporation, proposed for use with magnetic resonance imaging (MRI) to improve pancreatic duct visualization for the detection of duct abnormalities to enhance clinical decision making i...

 

Circulatory System Devices Panel of the Medical Devices AdComm

Meeting Date: 6/13/12-6/13/12  

On June 13, 2012, the committee will discuss, make recommendations and vote on information related to the premarket approval application for the Edwards SAPIEN Transcatheter Heart Valve sponsored by Edwards Lifesciences. The Edwards SAPIEN Transcatheter Heart Valve is indicated for use in patient...

Oncologic Drugs AdComm

Meeting Date: 6/20/12-6/20/12  

On June 20, 2012, during the morning session, the committee will discuss new drug application (NDA) 203213, with the established name semuloparin sodium injection, application submitted by sanofi-aventis U.S. LLC. The proposed indication (use) for this product is for the prophylaxis of venous thr...

General and Plastic Surgery Devices Panel of the Medical Devices AdComm

Meeting Date: 6/21/12-6/21/12  

On June 21, 2012, the committee will discuss, make recommendations, and vote on information related to the premarket approval application, sponsored by Dune Medical Devices, Inc., for the MarginProbe System, that utilizes electromagnetic waves to characterize human tissue in real time and provide...

Read more at http://www.fdalive.com/index.cfm 

  

Former FDA Commissioner Offers Path Toward a 21st-Century FDA

Former commissioner of the Food and Drug Administration Andrew von Eschenbach (2005-2009) recently wrote an op-ed article for the Wall Street Journal outlining actions which he believes will strengthen the FDA and make a more efficient path for approval of new biopharmaceutical drugs and treatments.  

In this article Eschenbach, currently the chairman of the Manhattan Institute's Project FDA, points to a lack of sufficient funding of the FDA which he claims made it difficult for the agency to procure "scientific tools and resources to review complex innovations more expeditiously and pioneer regulatory pathways for state-of-the-art therapies that defy current agency conventions." These deficits, he argues, has halted the approval of treatments as the FDA scrambled to keep up with new science and techniques.  

He opined,

"The FDA isn't obstructing progress because its employees are mean-spirited or foolish. But for decades, Congress has starved the agency of critical funding, limiting its scientists' ability to keep up with peers in private industry and academia. The result is an agency in which science-based regulation often lags far behind scientific discovery. This forces the FDA to slow the approval of new treatments—and at times creates acrimonious litigation between the FDA and innovators, not to mention disillusionment among desperate patients."

To alleviate this problem, Eschenbach calls for increased funding of the FDA as well as greater collaboration between this agency and other leaders in the field, such as physicians and companies currently developing new technologies and techniques.  By working in tandem, Eschenbach feels the FDA would establish greater understanding of new science and be in a better position to more efficiently and accurately evaluate breakthroughs.    

Read the full article here.  

FDA Starts scrutinizing how adipose tissue is used @ Intellicel Bio - $SVFC

Judging by a recent "warning letter" issued to the CEO of Intellicell Biosciences (OTCQB: SVFC), the FDA is getting stricter regarding the processing of adipose tissue. 

 

The letter, which was posted on the FDA website, regards Intellicell's processing of adipose tissue for use in orthopedic therapies. Intellicell is a company processing adipose tissue, claiming their “ultrasonic cavitation” technology is only minimally manipulated; hence they fall under less FDA scrutiny (ie, no FDA approval required).

It seems that the FDA is concerned about how this specific process may be changing the structural characteristics of the adipose tissue (fat) itself. It appears that regardless of the process the FDA seems to be considering the product as a drug. 

 

The letter includes the following excerpt:

"IntelliCell’s adipose derived stem cells are human cells, tissues, or cellular and tissue-based products (HCT/Ps) as defined in 21 CFR 1271.3(d). However, this cellular product does not meet all of the criteria in 21 CFR 1271.10(a) and therefore is not regulated solely under section 361 of the Public Health Service Act (PHS Act) [42 U.S.C. 264] and the regulations in 21 CFR Part 1271. Specifically, your processing alters the relevant characteristics of the adipose tissue relating to the tissue’s utility for reconstruction, repair, or replacement. Therefore the processing would not meet the definition of minimal manipulation for structural tissue such as adipose tissue. As a result, the IntelliCell product does not meet the criterion in 21 CFR 1271.10(a)(1)." 

See the full letter on fda.gov.  

FDA Declared Support for a New AntioBiotic Pathway - Positive for Companies like PolyMedix ($PYMX)

News from the FDA which declared its oprganizational support for a new pathway for review and approval of drugs to treat infections that are not susceptible to existing drugs said Janet Woodcock, director of FDA's Center for Drug Evaluation and Research. The new pathway was proposed by the Infectious Diseases Society of America in a statement submitted to the House Energy and Commerce Committee's health subcommittee. Under the proposal, products designated as "Special Population Limited Medical Use Drugs" could be studied in abbreviated clinical trials for specific populations, such as patients with drug-resistant infections, and measures would be taken to limit off-label use.

Speaking at a health subcommittee hearing Thursday, Woodcock also endorsed the goals of the Faster Access to Specialized Treatments (FAST) Act, which seeks to expand FDA's use of accelerated approvals. She said legislation instructing FDA to establish a new pathway for reviewing breakthrough drugs would be "helpful".

Daily Dose Conclusion: This news is positive for names like Polymedix (PYMX).

Flash Player 9 or higher is required to view the chart
Click here to download Flash Player now

View the full PYMX chart at Wikinvest

Athersys ($ATHX) : GvHD Phase 1 data is encouraging...

Athersys (NASDAQ: ATHX) announced in their press release positive results from its Phase I clinical trial of MultiStem, its cell therapy product. The trial was administered to individuals undergoing allogeneic hematopoietic stem cell transplants (HSCT) for the treatment of leukemia and related conditions.

Data highlights from the study include:

• The majority of patients participating in the study received transplants from unrelated donors (19 of 36), and nearly all of the patients received peripheral blood stem cell (PBSC) transplants (34 of 36), both of which are associated with a higher risk of GvHD;
• All patients experienced successful neutrophil engraftment (median time of engraftment 15 days), and 86% of patients experienced successful platelet engraftment (median time of engraftment 16 days) which compares favorably to historical clinical experience for this patient population demonstrating a positive impact on blood and immune system recovery;
• Substantial reduction in acute GvHD incidence, relative to historical experience, at the highest single dose (11% grade II-IV GvHD, and 0% grade III-IV GvHD).

These clinical results provide the foundation for further accelerated development of MultiStem for the prevention and reduction of GvHD. Following final review of the data, and subject to input from its key scientific and clinical advisers, Athersys plans to meet with the FDA to discuss plans for the next phase of clinical development. This could include a blinded, controlled phase II/III study of MultiStem for GvHD prophylaxis and HSCT support.

Daily Dose Conclusion: Athersys is building a solid foundation to expand the pipeline in a product with Multistem.

See all ATHX articles @ Daily Dose

Flash Player 9 or higher is required to view the chart
Click here to download Flash Player now

View the full ATHX chart at Wikinvest