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GCVRZ: Lemtrada FDA approval imminent

20 Sep

Disclamer: The author has a position in the Genzyme Contingent Value Rights (GCVRZ).

A few days ago, Lemtrada was approved by the regulatory authorities in Europe for the European market.

Regulatory agencies like the FDA are a little like the rating agencies in the financial world in that their decisions often mirror each other. Therefore, with this approval, I believe that the FDA approval of Lemtrada is now imminent.

Generally, I have found that European regulatory authorities are faster and more willing to take a risk with a new drug. So this is the case with Lemtrada which is approved first in Europe. With the approval of Lemtrada in Europe, this paves the way for FDA to approve it in the US as well.

Once FDA approves the drug, the second milestone payment of $1 will be triggered for the holders of GCVRZ.

I reproduce below the press release from Sanofi and the recommendations of the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) which gave rise to the approval.

European Commission Approves Genzyme’s Multiple Sclerosis Treatment Lemtrada™ (alemtuzumab)

  • Follows Recent European Commission Approval of Multiple Sclerosis Treatment Aubagio® (teriflunomide)
  • Approvals Set the Stage for Launches Throughout EU and Strongly Position Genzyme as a Committed Partner to the MS Community

Sanofi-Genzyme-logoParis, France – September 17, 2013 – Sanofi (EURONEXT: SAN and NYSE: SNY) and its subsidiary Genzyme announced today that the European Commission has granted marketing authorization for Lemtrada™. This follows the August 30th approval of Aubagio®. The company intends to begin launching both products in the EU soon.

“The approvals of Lemtrada and Aubagio in the European Union represent an important milestone for Genzyme and demonstrate our focus on scientific innovation and commitment to multiple sclerosis patients,” said Genzyme CEO and President, David Meeker, M.D. “This is particularly exciting as the EU approval is the first for Lemtrada globally. We look forward to making these unique therapies available to MS patients very soon.”

Lemtrada is indicated for the treatment of adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease defined by clinical or imaging features. Lemtrada 12 mg has a novel dosing and administration schedule of two annual treatment courses. The first treatment course of Lemtrada is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.

The Lemtrada clinical development program included two pivotal randomized Phase III studies comparing treatment with Lemtrada to high-dose subcutaneous interferon beta-1a (Rebif®) in patients with RRMS who had active disease and were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II), as well as an ongoing extension study. In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada vs. interferon beta-1a.

The most common side effects of Lemtrada are infusion associated reactions, infections (upper respiratory tract and urinary tract), lymphopenia and leukopenia. Serious autoimmune conditions can occur in patients receiving Lemtrada. A comprehensive risk management program will support early detection and management of these autoimmune events.

Aubagio 14 mg is a once-daily, oral therapy indicated for treatment of adult patients with RRMS. The EU approval was based on data from the Phase III TEMSO (TEriflunomide Multiple Sclerosis Oral) and TOWER (Teriflunomide Oral in people With relapsing remitting multiplE scleRosis) trials. The EU approval of Aubagio includes new active substance designation.

“Multiple sclerosis necessitates a highly individualized treatment approach, and the increasing diversity of options is good news,” said Hans-Peter Hartung M.D., Ph.D., Professor and Chairman of the Department of Neurology at Heinrich-Heine-University in Duesseldorf, Germany. “The Lemtrada clinical trial data support its potential to meaningfully address disability in active RRMS patients, while Aubagio’s efficacy, safety and convenient dosing may provide an important alternative to injectable therapies. The approvals of Lemtrada and Aubagio represent a significant step forward in the way we think about treating this disease.”

Multiple sclerosis is estimated to affect more than 2.1 million people globally. There are approximately 630,000 people affected by MS in Europe.

“This is a hopeful time for people with MS,” said John Golding, President of the European Multiple Sclerosis Platform. “These approvals demonstrate the great progress being made towards introducing more differentiated treatment options that address important unmet needs.”

FDA action on Genzyme’s supplemental Biologics License Application seeking U.S. approval of LemtradaTM (alemtuzumab) for the treatment of relapsing MS is expected in late 2013. Lemtrada is also under review by other regulatory agencies. Aubagio is approved to treat relapsing MS in the United States, Australia, Argentina, Chile, and South Korea, and is under review by additional regulatory agencies.

LEMTRADA has been in active clinical development for MS for more than 10 years. The clinical development program involved more than 1,700 patients.

EMANew

Summary of Opinion

27 June 2013
EMA/377379/2013
Committee for Medicinal Products for Human Use (CHMP)

On 27 June 2013, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Lemtrada 12 mg alemtuzumab in 1.2 ml (10 mg/ml) concentrate for solution for infusion intended for the treatment of relapsing remitting multiple sclerosis. The applicant for this medicinal product is Genzyme Therapeutics Ltd. They may request a re-examination of any CHMP opinion, provided they notify the European Medicines Agency in writing of their intention within 15 days of receipt of the opinion.

The approved indication is: “treatment of adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease defined by clinical or imaging features “.

Alemtuzumab is a humanized monoclonal antibody directed against the cell surface glycoprotein CD52. The mechanism by which alemtuzumab exerts its therapeutic effects in multiple sclerosis is not fully elucidated. However, research suggests immunomodulatory effects through depletion and repopulation of lymphocytes.

The benefits with Lemtrada are its ability to reduce the relapse rate and slow disability progression.

The most common side effects are infusion associated reactions (including headache, flushing, nausea, urticaria, rash, pruritus, pyrexia and fatigue), upper respiratory tract infection, urinary tract infection, lymphopenia and leukopenia. In addition, side effects pertaining to the thyroid gland (including overactive or under-active thyroid gland, or goitre and auto-immune conditions) were commonly observed in patients treated with alemtuzumab.

A pharmacovigilance plan for Lemtrada will be implemented as part of the marketing authorisation.

Treatment with Lemtrada should be initiated and supervised by a neurologist experienced in the treatment of patients with MS. Specialists and equipment required for the timely diagnosis and management of the most frequent adverse reactions, especially autoimmune conditions and infections, should be available.

Resources for the management of hypersensitivity and/or anaphylactic reactions should be available.

Patients treated with Lemtrada must be given the Patient Alert Card and Patient Guide and be informed about the risks of Lemtrada.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

The CHMP, on the basis of quality, safety and efficacy data submitted, considers there to be a favourable benefit-to-risk balance for Lemtrada and therefore recommends the granting of the marketing authorisation.

 
4 Comments

Posted by on September 20, 2013 in News, Portfolio, US Market

 

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4 responses to “GCVRZ: Lemtrada FDA approval imminent

  1. GW

    September 27, 2013 at 8:49 am

    Just repeating the comment I made on your earlier post where the milestone info was laid out, in case you miss it.

    Interesting article, as I hold this from the takeover. But in your schedule you write “FDA Approval $1 1H2013″.

    So unless this is a typo surely the deadline has already been missed? Now we are in 2H2013.

     
    • FuturesAsia

      September 27, 2013 at 12:59 pm

      Yes. You’re absolutely right. The FDA approval has indeed been delayed… I was expecting an approval in 1H2013 myself but it has been delayed.

       
    • FuturesAsia

      September 27, 2013 at 1:29 pm

      It now looks my estimate of the time Lemtrada will be approved is too aggressive. It is however, important to note that $1 will be paid ONLY if the approval of the drug occurs before 31 Mar 2014. If it occurs after that, then the value of the CVR will be much less… not that the $1 will not be paid eventually but the $1 will be paid only after sales hit $1.8 billion. Here is the actual wordings from the SEC document:

      The CVR contains 5 discrete milestones related to the development and commercialization of Lemtrada:

      1. Approval Milestone: CVR holders are entitled to receive $1 per CVR after U.S. FDA approval of Lemtrada for treatment of multiple sclerosis, if the approval occurs on or before March 31, 2014.
      2. Product Sales Milestone #1: CVR holders are entitled to receive $2 per CVR in the event net sales for Lemtrada total $400 million or more on a global basis during specified periods following product launch.
      3. Product Sales Milestone #2: CVR holders are entitled to receive $3 per CVR in the event global net sales for Lemtrada total $1.8 billion during any 4 consecutive calendar quarters. Any quarters used in the achievement of this sales milestone cannot be used again for the achievement of any subsequent milestones. In addition, if this Product Sales Milestone #2 is achieved despite U.S. FDA approval of Lemtrada for treatment of multiple sclerosis not having occurred on or before March 31, 2014 (and so the Approval Milestone Payment was not made), CVR holders will be entitled to receive an additional $1 per CVR for Product Sales Milestone #2.
      4. Product Sales Milestone #3: CVR holders are entitled to receive $4 per CVR in the event global net sales for Lemtrada total $2.3 billion during any 4 consecutive calendar quarters. Any quarters used in the achievement of this sales milestone cannot be used again for the achievement of any subsequent milestones.
      5. Product Sales Milestone #4: CVR holders are entitled to receive $3 per CVR in the event global net sales for Lemtrada total $2.8 billion during any 4 consecutive calendar quarters.

      http://www.sec.gov/Archives/edgar/data/732485/000095012311017162/b85162exv99waw43.htm

       
      • GW

        September 27, 2013 at 3:11 pm

        Many thanks for the clarification! These seem to be the salient points for now:

        March 31, 2014 is the current “date to beat” for $1, but there is a second chance at this $1 if $1.8b (over 4q) is sold subsequently.

        And $400m (1st y sales, staggered across EU, US etc) and $1.8b (any 4q) are the subsequent sales targets to unlock $2 and $3 respectively.

        Given the current price is $2, the market may be betting either 1) or 2)
        1) Mar 31 2014 will be missed but $400m will be sold in y1
        2) Mar 2014 missed, $400m y1 not sold, but 50% chance $1.8b (4q) sold eventually.

         

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