Full-Time
Develops oral drugs for rare diseases
No salary listed
North Carolina, USA
Remote
Must live in a Piedmont Region state: Virginia, North Carolina, South Carolina, Tennessee, Kentucky, or Georgia.
PharmD, PhD, MD
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BioCryst Pharmaceuticals develops oral small-molecule drugs aimed at treating rare diseases, with a focus on hereditary angioedema (HAE). The company uses traditional cell biology and medicinal chemistry to design synthetic compounds that target specific protein pathways involved in disease. Its products work as oral therapies that modulate disease-related proteins to reduce symptoms and improve daily functioning, progressing from discovery through clinical trials toward commercialization. BioCryst differentiates itself by concentrating on rare diseases and pursuing oral, non-biologic treatments, leveraging a combined science approach and a patient-focused strategy to bring therapies to market. Its goal is to deliver meaningful, clinically validated treatments that enable patients to engage more fully in everyday activities and improve their quality of life, supported by its drug pipeline and potential licensing opportunities.
Company Size
501-1,000
Company Stage
IPO
Headquarters
Durham, North Carolina
Founded
1986
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BCRX stock posts worst day in over 6 months after PHVS trial win in rare swelling disease. Published: Sep 09 2026, 05:00 AM IST * FB * TW * Linkdin * Whatsapp * GNFollow Us Pharvaris said on Tuesday its once-daily experimental pill cut monthly swelling attacks by 83% versus a dummy treatment in a late-stage study of hereditary angioedema patients. * BioCryst's Orladeyo is approved to prevent HAE attacks. * Orladeyo sales have continued to grow, and the company is guiding for $625 million to $645 million in revenue from the drug this year. * BioCryst CEO Charlie Gayer said last month that the company wants to buy rare-disease medicines after Orladeyo's success. Shares of BioCryst Pharmaceuticals (BCRX) fell sharply on Tuesday after a competitor reported strong late-stage results in hereditary angioedema, the rare swelling disorder that underpins BioCryst's main product. BCRX fell 9% on Tuesday and clocked its worst day since late February. A rival pill raises the bar. The pressure came from Pharvaris (PHVS), which said its once-daily experimental pill cut monthly swelling attacks by 83% compared with a dummy treatment in a late-stage study. In the two most common forms of the disease, attacks fell by 87%. The medicine was generally well tolerated, and Pharvaris plans to seek approval in the first half of 2027. PHVS shares jumped 7% in regular trading and another 3% after-hours. That market is the same one served by BioCryst's Orladeyo, an approved once-daily capsule used to prevent attacks. Investors marked down Orladeyo on the risk of a stronger oral rival after 2027. Orladeyo market. Orladeyo sales have continued to grow, and the company is guiding for $625 million to $645 million from the drug this year. A pellet version for young children has begun shipping after an earlier manufacturing delay. The drug has generated more than $2 billion in revenue since U.S. approval in 2020. BioCryst is also developing a second hereditary angioedema medicine, navenibart, with late-stage data still expected in 2027. BioCryst CEO Charlie Gayer said last month that the company wants to buy rare-disease medicines after Orladeyo's success. Gayer has said the next products should come from outside deals, not its own lab. Larger drugmakers have been chasing bigger assets. BioCryst is positioning itself as a buyer in the rare-disease gap they leave behind. How did BCRX retail traders react? On Stocktwits, retail sentiment around BCRX rose from 'bearish' to 'neutral' over the past 24 hours, while message volume jumped from 'low' to 'high' levels. A Stocktwits user dismissed Tuesday's selloff, noting that the company already has a real commercial product. "I'm really not worried... it will go up sooner than later... market trying to get rid of the weak hands," they said. View this Stocktwits post Another user opined that no one will switch out of Orladeyo even if Pharvaris's drug gets approved. It also noted that PHVS is valued higher than BCRX despite having no approved products. View this Stocktwits post BCRX stock has gained 16% year-to-date. For updates and corrections, email newsroom[at]stocktwits[dot]com. Stay updated with all the latest Business News, including market trends, Share Market News, stock updates, taxation, IPOs, banking, finance, real estate, savings, and investments. Track daily Gold Price changes, updates on DA Hike, and the latest developments on the 8th Pay Commission. Get in-depth analysis, expert opinions, and real-time updates to make informed financial decisions. Download the Asianet News Official App from the Android Play Store and iPhone App Store to stay ahead in business. 0 Comments / 0 New
CareMed has been selected as a national pharmacy partner by BioCryst and adds Orladeyo(R)(berotralstat) to growing Rare Disease portfolio. By GlobeNewswire September 9, 2026 NEW HYDE PARK, N.Y., Sept. 08, 2026 (GLOBE NEWSWIRE) - CareMed, an independent specialty pharmacy, has been selected as a pharmacy partner by BioCryst Pharmaceuticals for Orladeyo(R)(berotralstat), a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 years and older.1 "The addition of Orladeyo reinforces our long-term commitment to supporting rare and complex disease, highlights the strength of our specialty platform in delivering high-touch, patient-centric care across rare, cancer, and orphan disease communities, and further positions us as a trusted partner for patients, providers, and manufacturers," said Benito Fernandez, Chief Commercial Officer. About CareMed Specialty Pharmacy: CareMed is an independent Specialty Pharmacy affiliate of Onco360 and leading clinical support services company. CareMed is committed to bringing together the stakeholders involved in the rare and complex disease treatment process and serve the specialized needs of patients, providers, hospitals, manufacturers, health plans, and payers. CareMed is headquartered in New Hyde Park, New York, and is a specialty pharmacy brand of PharMerica Corporation, a leading institutional pharmacy, specialty infusion, and hospital services company servicing healthcare facilities in the United States. It is accredited through URAC, URAC Rare Disease, ACHC, and NAPB. For more information about CareMed, please visit CareMed.com. Media Contact: Benito Fernandez, Chief Commercial Officer References: 1Orladeyo(R)(berotralstat) [Package Insert]. Durham, NC. BioCryst Pharmaceuticals, Inc. 2025.
BioCryst Pharmaceuticals is leveraging profits from its hereditary angioedema drug Orladeyo to acquire rare disease treatments. The company expects up to $645 million in 2026 sales from Orladeyo, which has generated over $2 billion since its 2020 FDA approval. CEO Charlie Gayer said BioCryst became profitable last year and aims to maintain profitability whilst expanding beyond being a single-product company. The firm is now seeking external acquisitions rather than internal development, targeting assets with peak sales around $300 million. This shift reflects a broader trend where small and midsize biotech companies are stepping into rare disease drug development as large pharmaceutical firms increasingly focus on multibillion-dollar assets. BioCryst's shares have risen approximately 25% since the start of the year.
BioCryst Announces Approval of ORLADEYO for Pediatric HAE Patients. Wednesday, August 26, 2026 BioCryst Pharmaceuticals has announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved ORLADEYO(R)(berotralstat) for the prevention of hereditary angioedema (HAE) attacks in children aged 2 to under 12 years. The once-daily oral treatment is approved as a prophylactic therapy for paediatric patients with HAE caused by C1-inhibitor deficiency. The approval provides children in Japan with an oral option for long-term prevention of HAE attacks. HAE is a rare genetic condition that can cause sudden and potentially serious swelling in different parts of the body. Traditional preventive treatment for children has often involved injections, which can add to the treatment burden for patients and caregivers. ORLADEYO is available in a granule formulation for young children. The formulation can be taken directly with water or milk, or mixed with a spoonful of soft, non-acidic food, offering a simpler method of administration. The Japanese approval is supported by interim results from the APeX-P clinical trial, which is evaluating the pharmacokinetics, safety and efficacy of ORLADEYO in children aged 2 to under 12 years with HAE due to C1-inhibitor deficiency. The study is assessing long-term prophylaxis in paediatric patients. Interim findings showed that ORLADEYO was generally well tolerated and had a consistent safety profile across the age group. The treatment also produced early and sustained reductions in monthly HAE attack rates, with no new safety concerns identified. Nasopharyngitis was the most commonly reported treatment-emergent adverse event. BioCryst plans to launch ORLADEYO for children in Japan after the completion of the country's National Health Insurance reimbursement pricing process. OrphanPacific serves as BioCryst's partner in Japan and holds the marketing authorisation for the product. ORLADEYO is a once-daily oral plasma kallikrein inhibitor designed to prevent HAE attacks. It is approved for use in more than 45 countries. The capsule formulation was first approved in Japan in January 2021 for adults and children aged 12 years and older. In December 2025, the US Food and Drug Administration approved the oral pellet formulation of ORLADEYO for the prevention of HAE attacks in children aged 2 to under 12 years. BioCryst has also submitted applications for the paediatric indication to the European Medicines Agency and Health Canada, with further regulatory submissions planned in other markets. The latest approval marks the second regulatory approval for ORLADEYO in Japan and expands access to oral preventive treatment for younger children living with HAE.
BioCryst Pharmaceuticals has received approval from Japan's Ministry of Health, Labor and Welfare for ORLADEYO (berotralstat) as a prophylactic therapy for pediatric patients with hereditary angioedema (HAE) aged 2 to under 12 years. ORLADEYO is the first and only oral prophylactic therapy for HAE patients aged 2 and above. The granule formulation can be taken directly with water or milk, or sprinkled over soft food, reducing the treatment burden compared to traditional injections. The approval is based on interim results from the APeX-P clinical trial, the largest trial of long-term prophylaxis in pediatric HAE patients. Results showed the therapy was generally well tolerated with sustained reductions in monthly attack rates. BioCryst will launch ORLADEYO for children in Japan following completion of the National Health Insurance System reimbursement pricing process. OrphanPacific holds the marketing authorisation in Japan.