Calliditas Therapeutics AB announced on Friday (October 5) that the European Medicines Agency (EMA) has issued a positive opinion on the company’s drug, setanaxib in Alport Syndrome (AS).
The US Food and Drug Administration (FDA) has given orphan drug designation (ODD) to Redx’s Zelasudil for the potential treatment of idiopathic pulmonary fibrosis (IPF).
If new restrictions are placed on the FDA’s Accelerated Approval pathway, as many as two thirds of the treatments that use this pathway would never reach patients, according to research released at the BIO International Convention last week.
The Australian biotechnology company has been granted ODD status from the agency for Veyonda, a therapy intended for the treatment of soft-tissue sarcoma.
The recent Outsourcing-Pharma webinar hosted a trio of industry experts highlighting challenges, opportunities, and innovations in the rare disease realm.
During the Rare/Orphan Diseases, Special Patient Population webinar, a group of industry experts discussed challenges and opportunities faced in the field.
An executive from the rare disease technology solutions company discusses how forcing connections with patients can boost research into such conditions.
The company’s cancer drug, devimistat, has received orphan status from the agency for treating clear-cell sarcoma, pancreatic cancer, and other diseases.
FDA approves the third anti-TB drug in more than 40 years after the compound showed significantly higher success rates in patients with multi drug-resistant and extensively drug-resistant TB.
After Pharm-Olam conducted an orphan disease trial for therapy for a rare blood disorder, the European Commission granted it marketing authorization, and FDA is to give it priority review.
As aging populations grow and treatment paradigms shift, the industry must prepare for greater challenges in the rare disease clinical trial space, says CRO executive.
Ergomed is bolstering its orphan drug development services with its acquisition of PSR Group BV in a deal worth up to €5.7m ($6.69m) – which will position the company to be a “global player” in the area, says CEO.
Biomedical Systems has announced a strategic partnership with RadMD through which the companies will collaborate on clinical trials and augment their imaging capabilities.
Takeda’s development center in the US has agreed to partner with BioXcel, a cloud-based big data solution provider, to repurpose old medicines to help treat rare diseases.
As small biotech companies continue to cash in on new investments, IPOs and increasing approval rates of orphan and rare disease drugs, CROs are cashing in too and helping the companies navigate the regulatory and clinical landscape.
Recruiting patients with rare diseases for clinical trials could become easier if the US Congress passes a bill to allow patients on supplemental security income (SSI) benefits to receive compensation.
Amsterdam Molecular Therapeutics (AMT) lead candidate AMT 011, a
Lipoprotein Lipase (LPL) Deficiency treatment has received Orphan
Drug Designation from the US Food and Drug Administration (FDA).
