Important: New Login Process

We've updated our access and security system. The first time you log in, enter your work email, and we'll guide you through the process.

  • If your organization uses Single Sign-On (SSO), you will be redirected to your company's login portal.
  • If you log in with a username and password, you will be prompted to set a new password before accessing your account.

Go to the Login Page to get started.

We appreciate your cooperation during this transition. If you need assistance, please call +1 650-552-4224 or email support@biocentury.com.

BioCentury
ARTICLE | Regulation

Lenmeldy: an example of what it takes to get an ultrarare disease drug across the FDA finish line

A small dataset lacking a randomized control group can be enough when the efficacy signal is big and the unmet need severe

March 20, 2024 12:30 AM UTC
Updated on Mar 20, 2024 at 10:14 PM UTC

With scores of gene therapies for ultrarare diseases languishing in pipelines, FDA’s approval of Orchard’s metachromatic leukodystrophy gene therapy offers a case study in a path forward.

The Orchard Therapeutics unit of Kyowa Kirin Co. Ltd. (Tokyo:4151) announced Monday that FDA granted approval to atidarsagene autotemcel, under the brand name Lenmeldy, based on data from 37 patients, none of whom were treated in a randomized-controlled trial. Instead, the data were pieced together from two single-arm, open-label studies and a European expanded access program. All the patients were treated at one site, the Ospedale San Raffaele in Milan, Italy, and the results were compared with natural history. ...

Get Unlimited Access
Continue reading with a free trial.
Or Purchase This Article

BCIQ Company Profiles

Gsk plc

Kyowa Kirin Co. Ltd.

BCIQ Target Profiles

Arylsulfatase A (ARSA)