Eiger Bioが肺動脈高血圧症治療でウベニメクスの希少疾病用医薬品指定を受ける

AsiaNet 62685（1546）

【パロアルト（米カリフォルニア州）2015年11月30日PR Newswire＝共同通信JBN】Eiger BioPharmaceuticals, Inc. は30日、米国の食品医薬品局（DEA）がウベニメクスを肺動脈高血圧症（PAH）治療の希少疾病用医薬品に指定したと発表した。

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Eigerのジョアン・クアン最高医学責任者（CMO）は「FDAの希少製品開発室（OOPD）は、希少疾病の治療につながりそうな製品の評価・開発の進展に役立ち得る医薬品候補を認定し指定するため、提出された科学・臨床データを評価する。OOPDがウベニメクスをPAHの希少疾病用医薬品に指定したことは喜ばしい」と語った。

▽ウベニメクスについて

経口投与低分子化合物であるウベニメクスは、アミノペプチダーゼおよび炎症誘発メディエーターのLTB4の形成における関与段階を触媒する役割を果たす酵素であるロイコトリエンA4加水分解酵素（LTA4H）を二重阻害することが知られている。ウベニメクスは日本では、 成人の急性非リンパ性白血病の治療後、生存率を伸ばし寛解を維持するための化学療法剤と合わせて使用することを認められている。ウベニメクスは日本で25年余り使用されており、日本化薬からベスタチン（TM）のブランド名で市販されている。ウベニメクスは米国や欧州ではどのような症状にも使用が認められていない。

▽PAHについて

肺動脈高血圧症（PAH）はある種の高血圧症であり、肺や心臓右室の動脈に影響を及ぼす。PAHは、肺細動脈と呼ばれる肺の小さな動脈が狭窄し、あるいは閉塞もしくは損傷した時に発症する。この結果、血液が肺を通過するのが難しくなり、肺動脈内の血圧が上昇する。血圧が高まると、心臓の右心室は血液を肺に送り込むため懸命に働き、最後には心筋が衰弱して機能しなくなる。PAHは進行性の命を脅かす病気であり、米国、EU、日本で希少疾病用医薬品の範ちゅうの対象になっている。

▽希少疾病用医薬品の資格について

希少疾病用医薬品法に基づき、FDAは希少疾病、あるいは米国で一般的に20万人未満しかかからない病気の治療を目的とする医薬品候補を希少疾病用医薬品に指定できる。希少疾病用医薬品指定は、適正な臨床試験実施の税額控除、処方薬受益者負担法（PDUFA）に基づく費用控除、承認から7年間の販売独占権を含む各種の開発奨励措置の対象となる。希少疾病用医薬品資格は治療用に承認された適応症に限定して適用され、他の適応症には適用されない。

▽Eigerについて

Eigarは臨床段階のバイオ医薬品企業で、希少疾病治療用の新しい医薬品を市場化することに専念している。同社は満たされない医療ニーズが高く、治療のバイオロジーが明らかで、そのために有効な療法を緊急に必要とする疾患に対応する能力を持った十分に特性化された多様な臨床段階の医薬品候補ポートフォリオを持っている。

▽投資家向け問い合わせ先

Jim Shaffer,

Eiger Bio, Inc.,

+1-919-345-4256,

jshaffer@eigerbio.com

ソース：Eiger BioPharmaceuticals, Inc.

Eiger BioPharmaceuticals Granted Orphan Drug Status for Ubenimex in Pulmonary Arterial Hypertension

PR62685

PALO ALTO, Calif., Nov. 30, 2015 /PRNewswire=KYODO JBN/ --

   Eiger BioPharmaceuticals, Inc. today announced that the US Food and Drug

Administration (FDA) has granted Orphan Drug status to ubenimex for the

treatment of pulmonary arterial hypertension (PAH).

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   "The FDA Office of Orphan Products Development (OOPD) evaluates scientific

and clinical data submissions from sponsors to identify and designate drug

candidates that could potentially treat rare diseases to help advance the

evaluation and development of such products," said Joanne Quan, MD, Chief

Medical Officer at Eiger. "We are pleased with the OOPD's designation of orphan

drug status for ubenimex in PAH."

   About Ubenimex

   Ubenimex is a well-characterized, oral, small-molecule, dual-inhibitor of

aminopeptidase and leukotriene A4 hydrolase (LTA4H), the enzyme responsible for

catalyzing the committed step in the formation of the pro-inflammatory

mediator, LTB4. Ubenimex is approved in Japan as an adjunct to chemotherapy

agents to extend survival and to maintain remission after treatment for acute

non-lymphocytic leukemia in adults. Ubenimex has been used for over 25 years in

Japan and remains commercially available through Nippon Kayaku under the brand

name, Bestatin(TM). Ubenimex is not approved for any indication in the US or

Europe.

   About PAH

   Pulmonary Arterial Hypertension (PAH) is a type of high blood pressure that

affects the arteries in the lungs and the right side of the heart.  PAH begins

when tiny arteries in the lungs, called pulmonary arterioles, become narrowed,

blocked or destroyed.  This makes it harder for blood to flow through the

lungs, and raises pressure within the lungs' arteries.  As the pressure builds,

the heart's lower right chamber (right ventricle) must work harder to pump

blood through the lungs, eventually causing the heart muscle to weaken and

eventually fail.  PAH is a progressive, life-threatening illness and meets

criteria for Orphan Designation in the US, EU, and Japan.

   About Orphan Drug Status

   Under the Orphan Drug Act, the FDA may grant orphan drug designation to drug

candidates intended to treat a rare disease or condition that generally affects

fewer than 200,000 individuals in the United States. Orphan drug designation

qualifies the sponsor of the drug candidate for various development incentives,

which may include tax credits for qualified clinical testing, an exemption from

fees under the Prescription Drug User Fee Act (PDUFA), and a seven-year

marketing exclusivity period following approval. Orphan Drug status applies

specifically to the active moiety and the indication for which it is granted,

and is not applicable to other indications for that moiety.

   About Eiger

   Eiger is a clinical-stage biopharmaceutical company committed to bringing to

market products for the treatment of Orphan diseases.  The Company has built a

diverse, clinical-stage portfolio of product candidates with the potential to

address diseases for which the unmet medical need is high, the biology is clear

and an effective therapy is urgently needed.  

   Safe Harbor Statements

   Additional Information about the Proposed Merger between Celladon

Corporation and Eiger BioPharmaceuticals, Inc. and Where to Find It

   In connection with the previously disclosed proposed merger between Celladon

Corporation and Eiger BioPharmaceuticals, Inc., Celladon and Eiger intend to

file relevant materials with the Securities and Exchange Commission, or the

SEC, including a registration statement on Form S 4 that will contain a

prospectus and a joint proxy statement.  Investors and security holders of

Celladon and Eiger are urged to read these materials when they become available

because they will contain important information about Celladon, Eiger and the

proposed merger. Investors and security holders are urged to read the joint

proxy statement, prospectus and the other relevant materials when they become

available before making any voting or investment decision with respect to the

proposed merger.

   This communication shall not constitute an offer to sell or the solicitation

of an offer to sell or the solicitation of an offer to buy any securities, nor

shall there be any sale of securities in any jurisdiction in which such offer,

solicitation or sale would be unlawful prior to registration or qualification

under the securities laws of any such jurisdiction. No offering of securities

in connection with the proposed merger shall be made except by means of a

prospectus meeting the requirements of Section 10 of the Securities Act of

1933, as amended.

   CONTACT: Investors - Jim Shaffer,

            Eiger Bio, Inc.

            +1-919-345-4256

            jshaffer@eigerbio.com

   SOURCE:  Eiger BioPharmaceuticals, Inc.