---

POLICY & COMMITTEE WATCH - Building the Bio-economy

---

BIOTECanada Submits Feedback to CADTH Consultation on Therapeutic Review Framework

In response to CADTH’s call for feedback on the draft Therapeutic Review Framework launched on December 7th, 2011, BIOTECanada recently submitted its position on the process by which therapeutic reviews will be performed.

A therapeutic review is a review of the most recent available evidence in the public domain regarding a single drug, a drug class, or drug category, which generally has been available in the marketplace for some time. These reviews are generally undertaken to coincide with and thus inform a formulary submission review and listing recommendation. CADTH has completed two pilots on conducting therapeutic reviews on the topics of biologics for adults with rheumatoid arthritis and third-line therapy for patients with type two diabetes. The findings from the evaluation of the two pilots culminated in the development of the therapeutic review framework, which was subsequently released to the public for third-party input.

The submission focuses on three key areas:
•    disclosure of unpublished data;
•    stakeholder feedback to the draft therapeutic review report;
•    integration with the common drug review (CDR); and
•    introduction of an appeal mechanism to the framework.

In addition, BIOTECanada has requested greater involvement in the early development of new frameworks and processes introduced by CADTH. It is hoped that many of the concerns identified during the stakeholder consultation period would not have to be raised if the association and other stakeholders are included in the development of processes and frameworks in which new products and services are delivered by CADTH. 

If you would like a copy of BIOTECanada’s submission to CADTH on the draft Therapeutic Review Framework or have any questions about the content in the submission, please contact Gibril Muddei.

---

Proposal to merge Industrial and Agriculture Committees approved by Board of Directors

The proposal by the Industrial and Environmental Committee (IEC) and Agriculture and Nutrition Advisory Board (ANAB) to merge was approved by the Board and work is underway to make a new combined Industrial & Agricultural Committee (IAC). With this new committee, BIOTECanada will achieve efficiencies in carrying out the mandate of the association in the industrial and agriculture biotechnology space and eliminate overlap between the previous ANAB and IEC.
Plans and terms of reference will be finalized at a combined meeting to be held in the coming weeks.  A full plan will be presented to the Board at their Feb 29th meeting. If you are interested in participating on the new IAC or would like to learn more about the IAC’s mandate, please contact Gibril Muddei.

---

Subsequent Entry Biologics – Events on the Horizon

BIOTECanada’s stakeholder calendar serves the engagement goals of the SEB Task Force as they seek to broaden awareness and interaction with key audiences.

On January 26, 2012 Professor Dr. Francesco Locatelli, Lecco Italy and Dr. Jian Wang, Health Canada will present “ Subsequent Entry Biologics: An Overview of the Canada and International Regulatory Landscape” in Toronto.

The calendar is designed as a general resource to include potential events where the SEB Task Force may choose to participate.  Events listed serve as a general directory, reviewed on a regular basis by the Task Force to determine opportunities for participation. Visit the calendar for upcoming events.

Log into the members' section to visit the calendar for upcoming events

---

PUBLIC AFFAIRS

---

January is Alzheimer’s Awareness Month

One in 11 Canadians over the age of 65 currently has Alzheimer's disease or a related dementia.

This year alone, one Canadian develops dementia every five minutes. The number of people living with Alzheimer's disease or a related dementia is expected to more than double, reaching 1.1 million Canadians within 25 years. Today, 5.4 million Americans are living with the disease. By 2050 it is estimated as many as 16 million Americans will have Alzheimer's disease.

Allon is currently enrolling patients in a pivotal Phase 2/3 clinical trial evaluating davunetide as a potential treatment for progressive supranuclear palsy (PSP), a rapidly-progressing and fatal movement disorder with dementia which is often misdiagnosed as Parkinson’s or Alzheimer’s disease.  Allon reached agreement on a Special Protocol Assessment with the U.S. Food and Drug Administration, as well as Orphan Drug and Fast Track Status in the U.S.  Similarly, Allon has Orphan Status for davunetide in the EU.

Davunetide is derived from a naturally occurring neuroprotective brain protein known as activity dependent neuroprotective protein (ADNP). Allon’s human clinical and pre-clinical data suggest that davunetide works on microtubules, structures in the brain critical to communication between cells, and central to the tau pathway.  Davunetide has shown statistically significant impacts on memory, activities of daily living, and a biomarker of brain cell function and integrity.  Allon has extensive intellectual property protecting davunetide.

Cangene Corporation has signed a collaborative research agreement with the University of British Columbia (UBC) under which they will develop the work of Dr. Neil Cashman, Scientific Director of PrioNet Canada, Canada Research Chair in Neurodegeneration and Protein Misfolding at UBC, and a scientist at the Vancouver Coastal Health Research Institute, aimed at developing immune-based therapies for Alzheimer's disease.

"We're enthusiastic about this partnership with Dr. Cashman, who is a leader in protein misfolding and neurodegenerative diseases. The initial research results from Dr. Cashman's lab, while still at an early stage of development, show promise for development of a novel immunotherapy," says John Sedor, President and CEO of Cangene.

BIOTECanada congratulates all members working in the area of Alzheimer’s research.

---

Federal Government Budget 2012 Indications

With the federal budget anticipated by late February, BIOTECanada continues to bring member input to the attention of senior government officials. Recently members of the Emerging Companies Advisory Board wrote to Minister of State for Science and Technology in response to the findings of the R&D Review Panel. We have also been working with our colleagues form the Biotech Accord to coordinate responses to the Panel findings. Read more - member's section gr flash

Relevant background materials

BIOTECanada Budget 2012 Submission

BIOTECanada R&D Panel Submission

---

Ontario Ministry of Health and Long Term Care - New Template Letters

The Ontario Ministry of Health and Long Term Care issues new templates to confirm Current Patent Status and Letter of Product Confirmation for drug submissions for use effective immediately.

Until February 29, 2012, manufacturers using the old template will be given forty eight (48) hours to correct the submission to the new template. As of March 1, 2012, submitted letters not using the new template will be deemed incomplete.

For letter and templates log into the Members' Only section of the website. 

---

MEMBER RECOGNITION

---

Enobia acquires 1 Billion Deal 

Alexion Pharmaceuticals ended 2011 by announcing a deal to buy Enobia Pharma, and its hypophosphatasia drug, for up to $1.08 billion.
Alexion is offering $610 million in cash upon completion of the transaction and up to $470 million more upon achievement of various regulatory and sales milestones. The key attraction of Enobia, a private company based in Montreal, Canada and Cambridge, Massachusetts is ENB-0040 (asfotase alfa), which is in Phase II for hypophosphatasia, "an ultra-rare, life-threatening, genetic metabolic disease for which there are no approved treatment options".
HPP is characterised by defective bone mineralisation and impaired phosphate and calcium regulation leading to progressive damage to multiple vital organs. This includes destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function and respiratory failure. About 50% of infants with the disease do not survive past one year of age.
Alexion chief executive Leonard Bell said that asfotase alfa, which has orphan drug designation in the USA and Europe, has shown "very compelling Phase II clinical data in infants and juveniles with hypophosphatasia". He added that the acquisition of Enobia "is very well aligned with Alexion’s objective to develop and deliver life-transforming therapies for patients suffering with ultra-rare, severe and life-threatening disorders".
Alexion, which markets the blood disorder drug Soliris (eculizumab) for the treatment of atypical haemolytic uremic syndrome and paroxysmal nocturnal haemoglobinuria, intends to finance the acquisition through cash on hand and $300 million of committed bank debt.
www.alxn.com
www.enobia.com