Pharming Group's Leniolisib Gets CHMP Positive Opinion

Ticker: PHAR · Form: 6-K · Filed: 2025-04-23T00:00:00.000Z

Sentiment: bullish

Topics: regulatory-approval, pharmaceuticals, drug-development, CHMP

TL;DR

Pharming's APDS drug leniolisib got a green light from EU regulators (CHMP), paving way for potential approval.

AI Summary

Pharming Group N.V. announced on April 23, 2025, that it has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) regarding its Marketing Authorisation Application (MAA) for leniolisib. This positive opinion is a significant step towards potential approval by the European Commission for the treatment of activated PI3K-delta syndrome (APDS).

Why It Matters

This positive CHMP opinion brings Pharming Group closer to bringing a new treatment option to patients with APDS in Europe, potentially impacting the treatment landscape for this rare genetic disorder.

Risk Assessment

Risk Level: medium — While the CHMP opinion is positive, final approval from the European Commission is still pending, and market adoption will depend on various factors.

Key Players & Entities

Pharming Group N.V. (company) — Filer of the report and developer of leniolisib
leniolisib (drug) — The drug for which a positive opinion was received
Committee for Medicinal Products for Human Use (CHMP) (company) — Regulatory body that issued the positive opinion
European Commission (company) — Regulatory body that will make the final approval decision
April 23, 2025 (date) — Date of the press release and announcement

FAQ

What is the significance of the CHMP's positive opinion for leniolisib?

The positive opinion from the CHMP is a crucial recommendation for the European Commission to grant marketing authorisation for leniolisib for the treatment of activated PI3K-delta syndrome (APDS).

What condition does leniolisib treat?

Leniolisib is intended for the treatment of activated PI3K-delta syndrome (APDS), a rare primary immunodeficiency.

When was this announcement made?

The announcement was made on April 23, 2025, via a press release furnished as Exhibit 99.1 to the Form 6-K filing.

What is the next step after the CHMP's positive opinion?

The next step is for the European Commission to review the CHMP's recommendation and make a final decision on granting marketing authorisation for leniolisib.

What is Pharming Group N.V.'s role in this development?

Pharming Group N.V. is the company that developed leniolisib and submitted the Marketing Authorisation Application (MAA) to the European regulatory authorities.

From the Filing

0001828316-25-000021.txt : 20250423 0001828316-25-000021.hdr.sgml : 20250423 20250423105903 ACCESSION NUMBER: 0001828316-25-000021 CONFORMED SUBMISSION TYPE: 6-K PUBLIC DOCUMENT COUNT: 3 CONFORMED PERIOD OF REPORT: 20250423 FILED AS OF DATE: 20250423 DATE AS OF CHANGE: 20250423 FILER: COMPANY DATA: COMPANY CONFORMED NAME: Pharming Group N.V. CENTRAL INDEX KEY: 0001828316 STANDARD INDUSTRIAL CLASSIFICATION: PHARMACEUTICAL PREPARATIONS [2834] ORGANIZATION NAME: 03 Life Sciences EIN: 000000000 STATE OF INCORPORATION: P7 FISCAL YEAR END: 1231 FILING VALUES: FORM TYPE: 6-K SEC ACT: 1934 Act SEC FILE NUMBER: 001-39822 FILM NUMBER: 25859717 BUSINESS ADDRESS: STREET 1: DARWINWEG 24 CITY: LEIDEN STATE: P7 ZIP: 2333 CR BUSINESS PHONE: 31 (0)71 5247 400 MAIL ADDRESS: STREET 1: DARWINWEG 24 CITY: LEIDEN STATE: P7 ZIP: 2333 CR FORMER COMPANY: FORMER CONFORMED NAME: Pharming Group Group N.V. DATE OF NAME CHANGE: 20201014 6-K 1 pharmingreceivespositivere.htm 6-K Document UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 6-K REPORT OF FOREIGN PRIVATE ISSUER PURSUANT TO RULE 13a-16 OR 15d-16 UNDER THE SECURITIES EXCHANGE ACT OF 1934 For the Month of April 2025 Commission File Number: 001-39822 Pharming Group N.V. (Exact Name of Registrant as Specified in Its Charter) Darwinweg 24 2333 CR Leiden The Netherlands (Address of principal executive offices) Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F. Form 20-F ☒ Form 40-F ☐ Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ☐ Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ☐ Furnished as Exhibit 99.1 to this Report on Form 6-K is a press release of Pharming Group N.V., or the Company, dated April 23, 2025. EXHIBIT INDEX Exhibit No. Description 99.1 Pharming receives positive recommendation from NICE for Joenja®▼(leniolisib) as a treatment for APDS SIGNATURE Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized. Pharming Group N.V. By: /s/ Fabrice Chouraqui Name: Fabrice Chouraqui Title: CEO Date: April 23, 2025 For media and investors only Pharming receives positive recommendation from NICE for Joenja® ▼ (leniolisib) as a treatment for APDS Joenja® is the first and only medicine specifically for APDS to be reimbursed within the NHS Recommended for adult and pediatric patients 12 years of age and older with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) Leiden, the Netherlands, April 23, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the National Institute for Health and Care Excellence (NICE) has issued positive final guidance recommending Joenja® (leniolisib) for reimbursement and use within the National Health Service (NHS) in England and Wales for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. The NICE recommendation is based on the totality of the data, including the Phase III clinical trial evaluating leniolisib, an oral selective PI3Kδ inhibitor, in patients with APDS, a rare and progressive primary immunodeficiency. In the primary analysis, treatment with leniolisib led to significant improvements in immune deficiency and immune dysregulation compared to placebo. The Phase III trial demonstrated an improvement in clinical outcomes, which was maintained during longer-term treatment within the open-label extension trial. Fabrice Chouraqui, Chief Executive Officer of Pharming, said : “This po