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BioMarin's sale of voucher earned with approval of rare pediatric disease drug Brineura the fourth publicly disclosed voucher sale this year with a price tag between $125m and $150m.

 

 

priority_review_voucher

BioMarin Pharmaceutical Inc.'s latest priority review voucher sale is further evidence that the price for the coupons, which entitle the holder to a speedier US FDA review, is leveling off.

 

 

On Nov. 27, BioMarin announced plans to sell a rare pediatric disease priority review voucher to an undisclosed purchaser for a lump sum payment of $125m. BioMarin gained the voucher with FDA's April 27 approval of the Brineura (cerliponase alfa) for late infantile neuronal ceroid lipofuscinosis type 2, a form of Batten disease. (Also see "BioMarin's Brineura Approval Shows FDA's Open Door For Orphan Drugs" - Pink Sheet, 2 May, 2017.)

 

 

BioMarin's announcement marks the fourth publicly disclosed sale of a priority review voucher in 2017. While details around the sellers, purchasers and exact dollar figures have not always been released, all of the publicly disclosed sales in 2017 appear to have been in the $125m to $150m range. (See bar chart.)

 

 

That price range is well below the publicly disclosed high mark for voucher sales, AbbVie Inc.'s $350m purchase in mid-2015 of a voucher United Therapeutics Corp. earned with the approval of the high-risk neuroblastoma drug Unituxin (dinutuximab).

 

 

 

 

However, the 2017 sales range marks an 85%-122% premium to the $67.5m BioMarin earned with the July 2014 sale of its first priority review voucher. That coupon, awarded upon approval of BioMarin's Morquio A Syndrome treatment Vimizim (elosulfase alfa), was purchased by Regeneron Ireland and redeemed for a priority review of the company's PCSK9 inhibitor Praluent(alirocumab), which is partnered with Sanofi.

 

 

The Vimizim coupon was the first publicly disclosed sale of a priority review voucher and, in retrospect, appears to have set a low bar for future sales.

 

 

Notably, that inaugural sale did not trigger an antitrust review because the $67.5m price tag did not meet the "size of transaction" text, a BioMarin spokeswoman said. In contrast, the $125m sale of the Brineura voucher is subject to standard antitrust review for a transaction of a sufficient size, she said.

 

 

[Editor’s note: Jonathan Goldsmith, Associate Director of CDER’s Rare Disease Program, and Ron Cooper, CEO of Albireo Pharma, will discuss priority review vouchers during a session of the FDA/CMS Summit. The meeting, which also features fireside chats with FDA Commissioner Scott Gottlieb and CMS Administrator Seema Verma, will take place Dec. 5-6 in Washington, DC. To register, please visit the conference homepage.

 

 

 

'The Right Prices'

Duke University economist David Ridley told the Pink Sheet that the recent transactions of $125-$150m per voucher "are the right prices."

 

 

Ridley was lead author of a landmark 2006 Health Affairs article that led to the creation of the tropical disease voucher program in the FDA Amendments Act of 2007. The voucher program was extended to rare pediatric diseases in the FDA Safety and Innovation Act of 2012 and, most recently, to medical countermeasures in the 21st Century Cures Act, which was signed into law in December 2016.

 

 

Ridley asserts that sales prices were to were too high in 2015 and 2016 given the supply of rare pediatric vouchers. He points to a March 2016 Health Affairs paper in which he predicted that prices would fall. Priority Review Voucher Expansion Could Be Bad, Program Creator Says - Pink Sheet, 12 May, 2016.)

 

 

"If the voucher supply holds steady, I think the prices will too," Ridley said. "In our paper, we wrote that prices could fall even lower, but that was too pessimistic. That study was based on the voucher value for drugs launched between 2007 and 2009 which happened to be lean years for blockbuster launches given standard review."

 

 

He suggested that vouchers remain a good deal for the purchasers.

 

 

For example, he pointed to the recent FDA approval of ViiV Healthcare's HIV treatment Juluca, a once-daily, fixed-dose combination of ViiV's integrase strand transfer inhibitor Tivicay(dolutegravir) with Janssen Pharmaceutical Cos.' non-nucleoside reverse transcriptase inhibitor Edurant (rilpivirine). Viiv secured a priority review for Juluca thanks to a voucher it purchased for $130m earlier this year from an undisclosed seller.

 

 

"If ViiV’s HIV drug achieves peak sales of $5bn, then the value of the voucher, in terms of time value of money alone, will be more than $500m plus any competitive or longevity effects," Ridley said.

 

 

More Vouchers Out There For The Picking

 

To date, FDA has awarded 17 priority review vouchers – 12 under the rare pediatric disease program and five for the approval of tropical disease treatments.

 

 

Estimated or actual dollar figures have been publicly disclosed in connection with the sale of nine of those vouchers. In addition, two companies, Novartis AG and Janssen R&D LLC, have redeemed vouchers awarded on their own products. (See chart.)

 

 

FDA is currently reviewing at least two applications for which recently purchased vouchers have been redeemed: Gilead Sciences Inc.'s fixed-dose combination of bictegravir, a novel investigational integrase strand transfer inhibitor, and emtricitabine/tenofovir alafenamide for the treatment of HIV-1 infection; and Teva Pharmaceutical Industries Ltd.'s fremanezumab for preventive treatment of migraine.

 

 

Priority Review Vouchers

Company/Product

Approval Date

Disease (Voucher Type1)

Voucher Status

Redemption Outcome

Novartis

Coartem(artemether/lumefantrine)

4/7/2009

Malaria

(T)

Redeemed for priority review of gouty arthritis indication forIlaris(canakinumab)

FDA complete response letter announced 8/29/2011

Janssen R&D

Sirturo(bedaquiline)

12/28/2012

Multi-drug resistant tuberculosis

(T)

Redeemed for priority review ofTremfya(guselkumab)

Tremfya approved 7/13/2017

BioMarin

Vimizim(elosulfase alfa)

2/14/2014

Mucopolysaccharidosis type IVA (Morquio A syndrome)

(P)

Sale to Regeneron Ireland for $67.5m announced 7/30/2014

Redeemed forPraluent(alirocumab), approved 7/24/2015

Knight Therapeutics

Impavido(miltefosine)

3/19/2014

Leishmaniasis

(T)

Sale to Gilead for $125m announced in 11/19/ 2014

Redeemed forOdefsey(emtricitabine/rilpivirine/tenofovir alafenamide),approved 3/1/2016

United Therapeutics

Unituxin(dinutuximab)

3/10/2015

High-risk neuroblastoma

(P)

Sale to AbbVie for $350m announced 8/19/2015

 

Retrophin (acquired from Asklepion Pharmaceuticals)

Cholbam(cholic acid)

3/17/2015

Ultra-rare bile acid synthesis disorders

(P)

Sale to Sanofi for $245m announced 5/27/2015

Redeemed forSoliqua(insulin glargine/lixisenatide), approved 11/21/2016

Wellstat

Xuriden(uridine triacetate)

9/4/2015

Hereditary orotic aciduria

(P)

Sold to AstraZeneca under 9/2014 agreement (financial terms not disclosed)

 

Alexion

Strensiq(asfotase alfa)

10/23/2015

Perinatal-, infantile- and juvenile-onset hypophosphatasia

(P)

Company plans to use voucher for one of its own products

 

Alexion

Kanuma(sebelipase alfa)

12/8/2015

Lysosomal acid lipase deficiency

(P)

Company has not publicly disclosed plans

 

PaxVax Bermuda

Vaxchora(cholera vaccine, live, oral)

6/10/2016

Cholera vaccine

(T)

Believed to have been purchased by Gilead for less than $338m2

 

Sarepta

Exondys 51(eteplirsen)

9/19/2016

Duchenne muscular dystrophy

(P)

Sale to Gilead for $125m announced 2/21/20172

 

Biogen

Spinraza(nusinersen)

12/23/2016

Spinal muscular atrophy

(P)

Company has not publicly disclosed plans

 

Marathon Pharmaceuticals

Emflaza(deflazacort)

2/9/2017

Duchenne muscular dystrophy

(P)

Company has not publicly disclosed plans

 

BioMarin

Brineura(cerliponase alfa)

4/27/2017

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)

(P)

Sale to undisclosed company for $125m announced 11/27/2017

 

Chemo Research

Benznidazole

8/29/2017

Chagas disease

(T)

 

 

Novartis

Kymriah(tisagenlecleucel)

8/30/2017

B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse

(P)

 

 

Ultragenyx Pharmaceutical

Mepsevii(vestronidase alfa-vjbk)

11/15/2017

Mucopolysaccharidosis type VII (MPS VII)

(P)

Company announced plans to sell the voucher

 

 

1Tropical disease (T); Rare pediatric disease (P)

2On Aug. 10, 2017, Gilead announced redemption of a priority review voucher for a fixed-dose combination of bictegravir, a novel investigational integrase strand transfer inhibitor, and emtricitabine/tenofovir alafenamide, a dual-NRTI backbone, for the treatment of HIV-1 infection. The user fee goal date is Feb. 12, 2018.

*GlaxoSmithKline and Viiv Healthcare announced June 1, 2017 redemption of a priority review voucher, purchased for $130m, with the submission ofJuluca, a single-tablet regimen ofTivicay(dolutegravir) andEdurant(rilpivirine) for the maintenance treatment of HIV-1 infection. FDA approved Juluca Nov. 21, 2017.

**Teva spent up to $150m to acquire a priority review voucher in Q3 2017 and redeemed it for fremanezumab for preventive treatment of migraine; the BLA submission was announced 10/17/2017.

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