Korro Receives European Medicines Agency Orphan Drug Designation for KRRO-110
Korro Bio (Nasdaq: KRRO) has received orphan drug designation from the European Medicines Agency (EMA) for KRRO-110, its investigational treatment for Alpha-1 Antitrypsin Deficiency (AATD). This follows the FDA's orphan drug designation granted in March 2025.
KRRO-110 is currently being evaluated in the Phase 1/2a REWRITE clinical study, with an interim readout expected in H2 2025. The EMA designation provides significant benefits, including protocol assistance, reduced fees, and market exclusivity upon approval, and is granted to medicines treating conditions affecting fewer than 5 in 10,000 people in the EU.
Korro Bio (Nasdaq: KRRO) ha ottenuto la designazione di farmaco orfano dall'Agenzia Europea per i Medicinali (EMA) per KRRO-110, il suo trattamento sperimentale per la Deficienza di Alfa-1 Antitripsina (AATD). Questa designazione segue quella concessa dalla FDA a marzo 2025.
KRRO-110 è attualmente in fase di valutazione nello studio clinico di Fase 1/2a REWRITE, con una prima analisi intermedia prevista nella seconda metà del 2025. La designazione EMA offre importanti vantaggi, tra cui assistenza sul protocollo, riduzione delle tariffe e esclusività di mercato al momento dell'approvazione, ed è riservata ai farmaci destinati a malattie che colpiscono meno di 5 persone su 10.000 nell'UE.
Korro Bio (Nasdaq: KRRO) ha recibido la designación de medicamento huérfano por parte de la Agencia Europea de Medicamentos (EMA) para KRRO-110, su tratamiento en investigación para la Deficiencia de Alfa-1 Antitripsina (AATD). Esto sigue a la designación de medicamento huérfano otorgada por la FDA en marzo de 2025.
KRRO-110 se está evaluando actualmente en el estudio clínico de Fase 1/2a REWRITE, con resultados intermedios esperados en la segunda mitad de 2025. La designación de la EMA proporciona beneficios significativos, incluyendo asistencia en el protocolo, reducción de tarifas y exclusividad en el mercado tras la aprobación, y se concede a medicamentos para condiciones que afectan a menos de 5 personas por cada 10,000 en la UE.
Korro Bio (나스닥: KRRO)가 유럽 의약품청(EMA)으로부터 KRRO-110에 대해 희귀의약품 지정을 받았습니다. KRRO-110은 알파-1 안티트립신 결핍증(AATD)을 치료하기 위한 임상 시험 약물입니다. 이는 2025년 3월 FDA로부터 받은 희귀의약품 지정에 이은 것입니다.
KRRO-110은 현재 1/2a상 REWRITE 임상 시험에서 평가 중이며, 2025년 하반기에 중간 결과가 발표될 예정입니다. EMA 지정은 프로토콜 지원, 수수료 감면, 승인 시 시장 독점권 등 중요한 혜택을 제공하며, EU 내 인구 10,000명당 5명 미만이 영향을 받는 질환을 치료하는 약물에 부여됩니다.
Korro Bio (Nasdaq : KRRO) a obtenu la désignation de médicament orphelin de l'Agence européenne des médicaments (EMA) pour KRRO-110, son traitement expérimental pour la déficience en alpha-1 antitrypsine (AATD). Cette désignation fait suite à celle accordée par la FDA en mars 2025.
KRRO-110 est actuellement évalué dans l'étude clinique de phase 1/2a REWRITE, avec une première analyse intermédiaire attendue au second semestre 2025. La désignation EMA offre des avantages importants, notamment une assistance pour le protocole, des frais réduits et une exclusivité commerciale après approbation, et est accordée aux médicaments traitant des affections touchant moins de 5 personnes sur 10 000 dans l'UE.
Korro Bio (Nasdaq: KRRO) hat von der Europäischen Arzneimittel-Agentur (EMA) die Orphan-Drug-Zulassung für KRRO-110 erhalten, seine experimentelle Behandlung für Alpha-1-Antitrypsin-Mangel (AATD). Dies folgt auf die Orphan-Drug-Zulassung der FDA im März 2025.
KRRO-110 wird derzeit in der Phase 1/2a REWRITE klinischen Studie evaluiert, mit einem Zwischenbericht erwartet in der zweiten Hälfte des Jahres 2025. Die EMA-Zulassung bietet bedeutende Vorteile, darunter Protokollhilfe, reduzierte Gebühren und Marktexklusivität nach Zulassung, und wird für Medikamente vergeben, die Erkrankungen behandeln, die weniger als 5 von 10.000 Menschen in der EU betreffen.
- EMA orphan drug designation granted for KRRO-110, providing development incentives and market exclusivity
- Previous FDA orphan drug designation already secured in March 2025
- Phase 1/2a clinical trial is ongoing with interim results expected in H2 2025
- None.
Insights
Korro's KRRO-110 receives EMA orphan drug designation for AATD, adding to FDA designation and advancing clinical development timeline.
Korro Bio's achievement of European Medicines Agency (EMA) orphan drug designation for KRRO-110 represents a significant regulatory milestone for their RNA editing therapy targeting Alpha-1 Antitrypsin Deficiency (AATD). This designation follows the FDA orphan drug status granted in March 2025, creating a valuable dual-continent regulatory advantage.
The orphan designations provide Korro with substantial benefits including protocol assistance, reduced regulatory fees, and critically, market exclusivity upon approval - typically 10 years in Europe compared to 7 years in the US. These incentives significantly enhance KRRO-110's commercial potential while reducing development costs.
KRRO-110 utilizes Korro's RNA editing platform, which aims to modify disease-causing mutations at the RNA level rather than permanently altering DNA. For AATD, this approach could potentially correct the protein misfolding that leads to both liver damage and lung disease in patients.
The ongoing Phase 1/2a REWRITE clinical study with an interim readout expected in H2 2025 will be crucial in validating their approach. The company's description of KRRO-110 as having "best-in-class potential" suggests confidence in their preclinical data, though investors should watch for the clinical data to confirm translation to human subjects.
This regulatory progress demonstrates Korro's advancement in the competitive rare disease space where specialized treatments can command premium pricing and sustained revenue despite smaller patient populations.
CAMBRIDGE, Mass., July 21, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced today that it has been granted orphan drug designation by the European Medicines Agency (EMA) Committee, who adopted a positive opinion on KRRO-110, Korro’s investigational medicine for the treatment of Alpha-1 Antitrypsin Deficiency (AATD).
“The EMA orphan drug designation for KRRO-110 is a significant milestone for Korro and highlights an urgent need to bring innovative solutions to people with AATD seeking new, disease-modifying therapies,” said Kemi Olugemo, MD, Chief Medical Officer at Korro. “Backed by encouraging preclinical data, KRRO-110 has best-in-class potential for the treatment of AATD. This designation represents an important step toward bringing KRRO-110 to European patients.”
In March 2025, KRRO-110 received orphan drug designation from the U.S. Food and Drug Administration for the treatment of AATD. KRRO-110 is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD, and an interim readout is expected in the second half of 2025.
The EMA grants orphan designation status to medicines intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases affecting fewer than 5 in 10,000 people in the European Union, and the medicine must be of significant benefit to patients. Orphan drug designation provides companies with various development incentives, including protocol assistance, reduced regulatory fees, and market exclusivity once the medicine is approved.
About REWRITE
REWRITE is a Phase 1/2a two-part single and multiple dose-escalating clinical study that will evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including healthy adults and clinically stable AATD patients with the PiZZ genotype. Secondary and exploratory endpoints include pharmacokinetic and pharmacodynamic parameters that will guide optimal dose selection for later stage studies. Interim data from Part 1 (single ascending doses in healthy volunteers and individuals with AATD) is expected in the second half of 2025, and completion of the study is anticipated in 2026. For additional information about the REWRITE study, visit ClinicalTrials.gov (NCT06677307).
About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110
AATD is a genetic disorder most commonly caused by a single missense mutation (G-to-A) in the SERPINA1 gene. Affected adults experience pulmonary emphysema and/or hepatic cirrhosis, as well as end organ manifestations. KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s proprietary RNA editing platform, Oligonucleotide Promoted Editing of RNA (OPERA®). KRRO-110, a potential best-in-class compound based on preclinical data, is designed to co-opt an endogenous enzyme, Adenosine Deaminase Acting on RNA (ADAR), to edit the “A” variant on SERPINA1 RNA, repair an amino acid codon, and restore secretion of normal AAT protein. This repair of the endogenous protein has the potential to clear protein aggregates from within liver cells to create a potentially clinically differentiated benefit for liver function and to preserve lung function by providing an adequate amount of normal AAT protein.
About Korro
Korro is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases. Korro is generating a portfolio of differentiated programs that are designed to harness the body’s natural RNA editing process, enabling a precise yet transient single base edit. By editing RNA instead of DNA, Korro is expanding the reach of genetic medicines by delivering additional precision and tunability, which has the potential for increased specificity and improved long-term tolerability. Using an oligonucleotide-based approach, Korro expects to bring its medicines to patients by leveraging its proprietary platform with precedented delivery modalities, manufacturing know-how, and established regulatory pathways of approved oligonucleotide drugs. Korro is based in Cambridge, Massachusetts. For more information, visit korrobio.com.
Korro intends to use its Investor Relations website, LinkedIn, and X (Twitter) as means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor Korro’s Investor Relations website and follow @KorroBio on LinkedIn, and X (Twitter), in addition to following Korro’s press releases, SEC filings, public conference calls, presentations, and webcasts.
Forward-Looking Statements
Certain statements in this press release may constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements include, but are not limited to, express or implied statements regarding expectations, hopes, beliefs, intentions or strategies of Korro regarding the future including, without limitation, express or implied statements regarding: the timing of data readouts and completion of the Phase 1/2a REWRITE clinical trial; KRRO-110’s potential to treat both liver and lung manifestations of AATD; KRRO-110’s best-in-class potential; and realizing the incentives offered from the orphan drug designation of KRRO-110; among others. In addition, any statements that refer to projections, forecasts, or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “strive,” “would,” “aim,” “target,” “commit,” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that statement is not forward looking. Forward-looking statements are based on current expectations and assumptions that, while considered reasonable are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management’s control including risks of conducting a clinical study; risks associated with regulatory oversight of clinical studies, enrollment risks and risks of expanding to other jurisdictions along with other risks inherent in biopharmaceutical development; risks associated with pre-clinical studies and clinical studies; and other risks associated with obtaining regulatory approvals and protecting intellectual property; as well as risks associated with general economic conditions (including recent geopolitical uncertainty and potential supply chain disruptions due to changes in economic policy); and other risks and uncertainties indicated from time to time in Korro’s filings with the SEC, including Part I Item 1A. “Risk Factors” in Korro’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, or SEC, as such may be amended or supplemented by its other filings with the SEC. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements in this press release, which speak only as of the date they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by law, Korro does not undertake or accept any duty to release publicly any updates or revisions to any forward-looking statements to reflect any change in its expectations or in the events, conditions or circumstances on which any such statement is based. This press release does not purport to summarize all of the conditions, risks and other attributes of an investment in Korro.
Korro Bio Contact Information
Investors Contact:
IR@korrobio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
FAQ
What is the significance of EMA's orphan drug designation for Korro Bio's KRRO-110?
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Has KRRO-110 received any other regulatory designations besides the EMA orphan drug status?
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