News
08/13/15: CURx Pharmaceuticals has FTI End of Phase 2 meeting with the FDA and reaches agreement on Phase 3 clinical design 05/28/15 CURx receives financial Award for FTI development from the Cystic Fibrosis Foundation Therapeutics
09/09/14: CURx Pharmaceuticals, Inc. licenses Captisol enable Lamotrigine from Ligand Pharmaceuticals
SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announced it has expanded its global license agreement with CURx Pharmaceuticals, Inc. to also include the development and commercialization of Ligand’s Captisol-enabled™ Lamotrigine program. Under the terms of the expanded license, Ligand will be eligible to receive $22 million in potential milestone payments, revenue from the sales of Captisol, and tiered royalties on future net sales in the range of 4% to 7% for Captisol-enabled™ Lamotrigine. CURx will be responsible for all costs related to the program.
“This is Ligand’s second program licensed to CURx and is another example of the importance of our Captisol® formulation technology to enable new and highly differentiated programs. We are pleased that our relationship with CURx has expanded with the licensing of Captisol-enabled™ Lamotrigine, a product candidate targeting hospital-based seizures,” commented John Higgins
, President and Chief Executive Officer of Ligand. “Captisol-enabled™ Lamotrigine adds to our growing pipeline of over 100 partnerships.”
Dinu Sen
, CEO and founder of CURx, commented, “We are delighted to report expansion of our portfolio of development products to three, including our second Captisol-enabled™ anti-epileptic drug. Consistent with our development plans for Captisol-enabled Topiramate, our goal is to develop this IV formulation of lamotrigine as a bridging therapy for patients with epilepsy who are temporarily unable to take oral lamotrigine.”
About Captisol-Enabled™ Lamotrigine
Captisol-enabled™ Lamotrigine was specifically formulated by Ligand to provide an intravenous or intramuscular option for hospitalized patients unable to use oral lamotrigine. Lamictal® (lamotrigine) is approved as adjunctive therapy in patients with partial seizures, adjunctive treatment of generalized seizures of Lennox-Gastuat syndrome and maintenance treatment of Bipolar I disorder. Captisol-enabled™ Lamotrigine is currently in pre-clinical development. Lamictal® and Lamictal® XR™ are marketed by GlaxoSmithKline. Lamictal® lost patent exclusivity in 2009.
About Captisol®
Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella
at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled six FDA-approved products, including Onyx Pharmaceuticals’ Kyprolis®, Baxter International's Nexterone® and Merck’s Noxafil® IV. There are currently more than 50 Captisol-enabled products in development, including Lundbeck’s Carbella™, Spectrum Pharmaceuticals’ Captisol-enabled Melphalan and Melinta Therapeutics’ delafloxacin IV program.
About CURx Pharmaceuticals, Inc.
CURx Pharmaceuticals develops therapeutics to provide effective solutions to currently unmet needs of patients with chronic diseases. CURX has licensed three mid- to late-stage molecules that fulfill our criteria for further development. These criteria include sound scientific rationale, strong preclinical and clinical study results, defined regulatory pathway to approval and robust IP protection. CURx Pharmaceuticals’ lead clinical asset is Fosfomycin Tobramycin for Inhalation (FTI), which is being developed as a treatment for patients with Cystic Fibrosis. CURx is committed to serving patients with innovative and differentiated therapeutics that will improve their quality of life and clinical outcome.
About Ligand Pharmaceuticals
Ligand is a biopharmaceutical company with a business model that is based upon the concept of developing or acquiring royalty revenue generating assets and coupling them to a lean corporate cost structure. Ligand’s goal is to produce a bottom line that supports a sustainably profitable business. By diversifying our portfolio of assets across numerous technology types, therapeutic areas, drug targets and industry partners, we offer investors an opportunity to invest in the increasingly complicated and unpredictable pharmaceutical industry. In comparison to its peers, we believe Ligand has assembled one of the largest and most diversified asset portfolios in the industry with the potential to generate revenue in the future. These therapies address the unmet medical needs of patients for a broad spectrum of diseases including diabetes, hepatitis, muscle wasting, Alzheimer's disease, dyslipidemia, anemia, asthma and osteoporosis. Ligand’s Captisol platform technology is a patent protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Ligand has established multiple alliances with the world's leading pharmaceutical companies including GlaxoSmithKline, Onyx Pharmaceuticals (a subsidiary of Amgen Inc.), Merck, Pfizer, Baxter International, Eli Lilly & Co., Spectrum Pharmaceuticals and AstraZeneca. Please visit www.captisol.com for more information on Captisol. For more information on Ligand, please visit www.ligand.com. Follow Ligand on Twitter @Ligand_LGND.
Forward-Looking Statements
This news release contains forward-looking statements by Ligand that involve risks and uncertainties and reflect Ligand’s judgment as of the date of this release. These include statements regarding clinical development of Captisol-enabled Lamotrigine, possibility of commercial success, efficacy, potency, competitiveness and the strength of Ligand's product portfolio. Actual events or results may differ from our expectations. For example, there can be no assurance that Captisol-enabled Lamotrigine will progress through clinical development or receive required regulatory approvals within the expected timelines or at all, that further clinical trials will confirm any safety or other characteristics or profile, that there will be a market of any size for Captisol-enabled Lamotrigine or that Captisol-enabled Lamotrigine will be beneficial to patients or successfully marketed. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Ligand's stock price. Additional information concerning these and other risk factors affecting Ligand's business can be found in prior press releases available via www.ligand.com as well as in Ligand's public periodic filings with the Securities and Exchange Commission at www.sec.gov. Ligand disclaims any intent or obligation to update these forward-looking statements beyond the date of this release. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
Source: Ligand Pharmaceuticals Incorporated
Ligand Pharmaceuticals Incorporated
John Higgins, President and CEO
(858) 550-7500
investors@ligand.com
@Ligand_LGND
02/19/14: CURx Pharmaceuticals Announces License Agreement with Gilead Sciences
SAN DIEGO, Feb. 19, 2014 /PRNewswire/ -- CURx Pharmaceuticals today announced it has entered into a global license agreement with Gilead Sciences, Inc. for development of Fosfomycin:Tobramycin for Inhalation (FTI) to treat Pseudomonas aeruginosa lung infection in cystic fibrosis (CF) patients. FTI is ready for Phase III clinical trials, having successfully completed a Phase II trial in CF patients.
"This is a great acquisition for CURx Pharmaceuticals which enables us to expand our portfolio and also to provide another inhaled therapeutic to the cystic fibrosis community", commented Dinu Sen, CEO and founder of CURx. "Clinical trials in CF patients with Pseudomonas aeruginosa infection have demonstrated FTI's safety and efficacy. Future trials may pave the way for its use against a range of bacterial infections in CF - there is an unmet need for antibiotics with such characteristics."
"Lung infections continue to be a serious problem for individuals with cystic fibrosis," commented Dr. Drucy Borowitz, Director of the Cystic Fibrosis Center of Western New York. "If further studies confirm that (FTI) is safe and efficacious it will be a significant addition to improving the health of people with CF." Dr. Patrick Flume, Director of the Cystic Fibrosis Center at Medical University of South Carolina added "Currently we have two drugs approved in the US for use in CF patients, but there remains a need for more options. The CF community welcomes the development of new treatment options such as FTI."
About FTI
FTI is a combination of fosfomycin and tobramycin, antibiotics well characterized for safety and anti-bacterial activity. FTI is formulated as a liquid for inhalation with an aerosol device. In Phase II clinical trials FTI was shown to be safe and to effectively maintain improvement in lung function achieved with Cayston in CF patients with Pseudomonas aeruginosa infection. FTI has demonstrated antibiotic activity against multiple pathogenic bacteria, including methicillin-resistant Staphylococcus aureus(MRSA), in preclinical studies.
About cystic fibrosis
Cystic fibrosis is a hereditary disease affecting the lungs and digestive system of 30,000 children and adults in the US and 70,000 worldwide. Chronic bacterial infections in the lungs of CF patients are common and can be life threatening if not adequately treated. Pseudomonas aeruginosa infects the lungs of half of all CF patients, while methicillin-resistantStaphylococcus aureus (MRSA) infects the lungs of about a quarter of CF patients.
About CURx Pharmaceuticals
San Diego-based CURx Pharmaceuticals specializes in developing late-stage therapeutics with high medical need. In August 2013 CURx Pharmaceuticals licensed intravenous topiramate from Ligand Pharmaceuticals, Inc. for treatment of hospitalized epilepsy patients unable to take oral medication. Please visit www.curxpharma.com for more information.
Forward-Looking Statements
This news release contains forward-looking statements. These statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. Factors that might cause such a difference include, among others, the completion of clinical trials, the FDA and other foreign review processes and other governmental regulation, CURx Pharmaceuticals' ability to successfully develop and commercialize drug candidates, competition from other pharmaceutical companies, the ability to effectively market products, and other factors. CURx Pharmaceuticals undertakes no duty to update forward-looking statements.
08/14/13: Ligand Enters Global License Agreement with CURx Pharmaceuticals, Inc. for Captisol-EnabledTM Topiramate Injection
SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announces it has entered a global license agreement with CURx Pharmaceuticals, Inc. for the development and commercialization of Ligand’s Captisol-enabled™ Topiramate Injection for the treatment of partial onset or primary generalized tonic-clonic seizures in hospitalized epilepsy patients who are unable to take oral topiramate. Under the terms of the agreement, Ligand will be eligible to receive more than $21 million in potential net milestone payments and net royalties on future sales of 6.0% to 7.5%.
“This is another great deal for Ligand and our Captisol technology, pairing our proven formulation agent with an approved medicine to create a potentially novel, important and highly differentiated drug,” commented John Higgins, President and Chief Executive Officer of Ligand. “We are very pleased to be entering this agreement. CURx is highly motivated to advance development with current plans calling for a Phase II trial to commence in 2014. This deal represents our latest addition to our growing pipeline of partnerships and another potentially lucrative shot-on-goal.”
Dinu Sen, Chief Executive Officer and founder of CURx, said of this transaction, “We are delighted to sign a licensing agreement with Ligand and plan to bring Captisol-enabled Topiramate Injection to our patients as soon as possible. In Phase I trials in healthy volunteers and patients at the University of Minnesota, the product demonstrated a faster onset of action than the orally administered drug. Our market research indicates there to be a tremendous need for a product with these characteristics.”
07/26/13: Ligand Receives Orphan Designation for Captisol-EnabledTM Topiramate Injection
SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for its proprietary Captisol-enabled™ Topiramate Injection for the treatment of partial onset or primary generalized tonic-clonic seizures in hospitalized epilepsy patients who are unable to take oral topiramate.
“The granting of orphan designation for Ligand’s Captisol-enabled Topiramate program is an important step in the future of this potentially life-saving therapeutic, and should provide an additional layer of market exclusivity for the program,” commented Matthew W. Foehr, Executive Vice President and Chief Operating Officer of Ligand. “In Phase 1 trials in healthy volunteers and patients at theUniversity of Minnesota, this product demonstrated a faster onset of action than the orally administered drug. Our goal is to find a committed partner to further progress the clinical development of this asset and add to our portfolio of fully-funded programs.”
“An injectable formulation of Topiramate will provide patients and clinicians with an important new product that ensures continuity of therapy and offers the potential for use in acute management of several neurological disorders,” declared Jim Cloyd, Pharm.D., Lawrence C. Weaver Endowed Chair in Orphan Drug Development, Professor of Experimental and Clinical Pharmacology and Director of the Center for Orphan Drug Research at the University of Minnesota-College of Pharmacy.
Orphan-drug designation is granted to drugs and biologics defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announced it has expanded its global license agreement with CURx Pharmaceuticals, Inc. to also include the development and commercialization of Ligand’s Captisol-enabled™ Lamotrigine program. Under the terms of the expanded license, Ligand will be eligible to receive $22 million in potential milestone payments, revenue from the sales of Captisol, and tiered royalties on future net sales in the range of 4% to 7% for Captisol-enabled™ Lamotrigine. CURx will be responsible for all costs related to the program.
“This is Ligand’s second program licensed to CURx and is another example of the importance of our Captisol® formulation technology to enable new and highly differentiated programs. We are pleased that our relationship with CURx has expanded with the licensing of Captisol-enabled™ Lamotrigine, a product candidate targeting hospital-based seizures,” commented John Higgins
, President and Chief Executive Officer of Ligand. “Captisol-enabled™ Lamotrigine adds to our growing pipeline of over 100 partnerships.”
Dinu Sen
, CEO and founder of CURx, commented, “We are delighted to report expansion of our portfolio of development products to three, including our second Captisol-enabled™ anti-epileptic drug. Consistent with our development plans for Captisol-enabled Topiramate, our goal is to develop this IV formulation of lamotrigine as a bridging therapy for patients with epilepsy who are temporarily unable to take oral lamotrigine.”
About Captisol-Enabled™ Lamotrigine
Captisol-enabled™ Lamotrigine was specifically formulated by Ligand to provide an intravenous or intramuscular option for hospitalized patients unable to use oral lamotrigine. Lamictal® (lamotrigine) is approved as adjunctive therapy in patients with partial seizures, adjunctive treatment of generalized seizures of Lennox-Gastuat syndrome and maintenance treatment of Bipolar I disorder. Captisol-enabled™ Lamotrigine is currently in pre-clinical development. Lamictal® and Lamictal® XR™ are marketed by GlaxoSmithKline. Lamictal® lost patent exclusivity in 2009.
About Captisol®
Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella
at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled six FDA-approved products, including Onyx Pharmaceuticals’ Kyprolis®, Baxter International's Nexterone® and Merck’s Noxafil® IV. There are currently more than 50 Captisol-enabled products in development, including Lundbeck’s Carbella™, Spectrum Pharmaceuticals’ Captisol-enabled Melphalan and Melinta Therapeutics’ delafloxacin IV program.
About CURx Pharmaceuticals, Inc.
CURx Pharmaceuticals develops therapeutics to provide effective solutions to currently unmet needs of patients with chronic diseases. CURX has licensed three mid- to late-stage molecules that fulfill our criteria for further development. These criteria include sound scientific rationale, strong preclinical and clinical study results, defined regulatory pathway to approval and robust IP protection. CURx Pharmaceuticals’ lead clinical asset is Fosfomycin Tobramycin for Inhalation (FTI), which is being developed as a treatment for patients with Cystic Fibrosis. CURx is committed to serving patients with innovative and differentiated therapeutics that will improve their quality of life and clinical outcome.
About Ligand Pharmaceuticals
Ligand is a biopharmaceutical company with a business model that is based upon the concept of developing or acquiring royalty revenue generating assets and coupling them to a lean corporate cost structure. Ligand’s goal is to produce a bottom line that supports a sustainably profitable business. By diversifying our portfolio of assets across numerous technology types, therapeutic areas, drug targets and industry partners, we offer investors an opportunity to invest in the increasingly complicated and unpredictable pharmaceutical industry. In comparison to its peers, we believe Ligand has assembled one of the largest and most diversified asset portfolios in the industry with the potential to generate revenue in the future. These therapies address the unmet medical needs of patients for a broad spectrum of diseases including diabetes, hepatitis, muscle wasting, Alzheimer's disease, dyslipidemia, anemia, asthma and osteoporosis. Ligand’s Captisol platform technology is a patent protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Ligand has established multiple alliances with the world's leading pharmaceutical companies including GlaxoSmithKline, Onyx Pharmaceuticals (a subsidiary of Amgen Inc.), Merck, Pfizer, Baxter International, Eli Lilly & Co., Spectrum Pharmaceuticals and AstraZeneca. Please visit www.captisol.com for more information on Captisol. For more information on Ligand, please visit www.ligand.com. Follow Ligand on Twitter @Ligand_LGND.
Forward-Looking Statements
This news release contains forward-looking statements by Ligand that involve risks and uncertainties and reflect Ligand’s judgment as of the date of this release. These include statements regarding clinical development of Captisol-enabled Lamotrigine, possibility of commercial success, efficacy, potency, competitiveness and the strength of Ligand's product portfolio. Actual events or results may differ from our expectations. For example, there can be no assurance that Captisol-enabled Lamotrigine will progress through clinical development or receive required regulatory approvals within the expected timelines or at all, that further clinical trials will confirm any safety or other characteristics or profile, that there will be a market of any size for Captisol-enabled Lamotrigine or that Captisol-enabled Lamotrigine will be beneficial to patients or successfully marketed. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Ligand's stock price. Additional information concerning these and other risk factors affecting Ligand's business can be found in prior press releases available via www.ligand.com as well as in Ligand's public periodic filings with the Securities and Exchange Commission at www.sec.gov. Ligand disclaims any intent or obligation to update these forward-looking statements beyond the date of this release. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
Source: Ligand Pharmaceuticals Incorporated
Ligand Pharmaceuticals Incorporated
John Higgins, President and CEO
(858) 550-7500
investors@ligand.com
@Ligand_LGND
02/19/14: CURx Pharmaceuticals Announces License Agreement with Gilead Sciences
SAN DIEGO, Feb. 19, 2014 /PRNewswire/ -- CURx Pharmaceuticals today announced it has entered into a global license agreement with Gilead Sciences, Inc. for development of Fosfomycin:Tobramycin for Inhalation (FTI) to treat Pseudomonas aeruginosa lung infection in cystic fibrosis (CF) patients. FTI is ready for Phase III clinical trials, having successfully completed a Phase II trial in CF patients.
"This is a great acquisition for CURx Pharmaceuticals which enables us to expand our portfolio and also to provide another inhaled therapeutic to the cystic fibrosis community", commented Dinu Sen, CEO and founder of CURx. "Clinical trials in CF patients with Pseudomonas aeruginosa infection have demonstrated FTI's safety and efficacy. Future trials may pave the way for its use against a range of bacterial infections in CF - there is an unmet need for antibiotics with such characteristics."
"Lung infections continue to be a serious problem for individuals with cystic fibrosis," commented Dr. Drucy Borowitz, Director of the Cystic Fibrosis Center of Western New York. "If further studies confirm that (FTI) is safe and efficacious it will be a significant addition to improving the health of people with CF." Dr. Patrick Flume, Director of the Cystic Fibrosis Center at Medical University of South Carolina added "Currently we have two drugs approved in the US for use in CF patients, but there remains a need for more options. The CF community welcomes the development of new treatment options such as FTI."
About FTI
FTI is a combination of fosfomycin and tobramycin, antibiotics well characterized for safety and anti-bacterial activity. FTI is formulated as a liquid for inhalation with an aerosol device. In Phase II clinical trials FTI was shown to be safe and to effectively maintain improvement in lung function achieved with Cayston in CF patients with Pseudomonas aeruginosa infection. FTI has demonstrated antibiotic activity against multiple pathogenic bacteria, including methicillin-resistant Staphylococcus aureus(MRSA), in preclinical studies.
About cystic fibrosis
Cystic fibrosis is a hereditary disease affecting the lungs and digestive system of 30,000 children and adults in the US and 70,000 worldwide. Chronic bacterial infections in the lungs of CF patients are common and can be life threatening if not adequately treated. Pseudomonas aeruginosa infects the lungs of half of all CF patients, while methicillin-resistantStaphylococcus aureus (MRSA) infects the lungs of about a quarter of CF patients.
About CURx Pharmaceuticals
San Diego-based CURx Pharmaceuticals specializes in developing late-stage therapeutics with high medical need. In August 2013 CURx Pharmaceuticals licensed intravenous topiramate from Ligand Pharmaceuticals, Inc. for treatment of hospitalized epilepsy patients unable to take oral medication. Please visit www.curxpharma.com for more information.
Forward-Looking Statements
This news release contains forward-looking statements. These statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. Factors that might cause such a difference include, among others, the completion of clinical trials, the FDA and other foreign review processes and other governmental regulation, CURx Pharmaceuticals' ability to successfully develop and commercialize drug candidates, competition from other pharmaceutical companies, the ability to effectively market products, and other factors. CURx Pharmaceuticals undertakes no duty to update forward-looking statements.
08/14/13: Ligand Enters Global License Agreement with CURx Pharmaceuticals, Inc. for Captisol-EnabledTM Topiramate Injection
SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announces it has entered a global license agreement with CURx Pharmaceuticals, Inc. for the development and commercialization of Ligand’s Captisol-enabled™ Topiramate Injection for the treatment of partial onset or primary generalized tonic-clonic seizures in hospitalized epilepsy patients who are unable to take oral topiramate. Under the terms of the agreement, Ligand will be eligible to receive more than $21 million in potential net milestone payments and net royalties on future sales of 6.0% to 7.5%.
“This is another great deal for Ligand and our Captisol technology, pairing our proven formulation agent with an approved medicine to create a potentially novel, important and highly differentiated drug,” commented John Higgins, President and Chief Executive Officer of Ligand. “We are very pleased to be entering this agreement. CURx is highly motivated to advance development with current plans calling for a Phase II trial to commence in 2014. This deal represents our latest addition to our growing pipeline of partnerships and another potentially lucrative shot-on-goal.”
Dinu Sen, Chief Executive Officer and founder of CURx, said of this transaction, “We are delighted to sign a licensing agreement with Ligand and plan to bring Captisol-enabled Topiramate Injection to our patients as soon as possible. In Phase I trials in healthy volunteers and patients at the University of Minnesota, the product demonstrated a faster onset of action than the orally administered drug. Our market research indicates there to be a tremendous need for a product with these characteristics.”
07/26/13: Ligand Receives Orphan Designation for Captisol-EnabledTM Topiramate Injection
SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for its proprietary Captisol-enabled™ Topiramate Injection for the treatment of partial onset or primary generalized tonic-clonic seizures in hospitalized epilepsy patients who are unable to take oral topiramate.
“The granting of orphan designation for Ligand’s Captisol-enabled Topiramate program is an important step in the future of this potentially life-saving therapeutic, and should provide an additional layer of market exclusivity for the program,” commented Matthew W. Foehr, Executive Vice President and Chief Operating Officer of Ligand. “In Phase 1 trials in healthy volunteers and patients at theUniversity of Minnesota, this product demonstrated a faster onset of action than the orally administered drug. Our goal is to find a committed partner to further progress the clinical development of this asset and add to our portfolio of fully-funded programs.”
“An injectable formulation of Topiramate will provide patients and clinicians with an important new product that ensures continuity of therapy and offers the potential for use in acute management of several neurological disorders,” declared Jim Cloyd, Pharm.D., Lawrence C. Weaver Endowed Chair in Orphan Drug Development, Professor of Experimental and Clinical Pharmacology and Director of the Center for Orphan Drug Research at the University of Minnesota-College of Pharmacy.
Orphan-drug designation is granted to drugs and biologics defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.